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The discussions highlight groundbreaking achievements in gene editing, particularly the case of a baby named KJ who received a personalized CRISPR-based therapy. Posts use strong phrases like 'groundbreaking' and 'milestone in personalized therapies' to convey excitement, while others question the ethics of such genetic interventions, demonstrating an engaging and reflective tone that underscores the importance of this medical advancement.
A majority of the digital discussions strongly endorse the advancements and capabilities of personalized gene editing technologies.
The posts reflect a very high level of curiosity and excitement about the possibilities of gene editing in medicine.
Numerous posts delve deeply into the specifics of the study, discussing methodologies and implications for future treatments.
Participants perceive this development as having a significant influence on the future of medical treatment for rare genetic diseases.
Brief Report: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/EtnnBhGfqi Editorial: Progress in the Development of N-of-1 Therapy https://t.co/YTdlxTQ7oN Science behind the Study: Personalized Gene Editing to Treat an Inborn Error of https://t.co/Vc2po1n8aB
A lipid nanoparticle–delivered base-editing therapy was custom designed for an infant with a urea-cycle disorder. The affected infant was treated at approximately 7 and 8 months of age. Read the Brief Report: https://t.co/EtnnBhGfqi Editorial: Progress in the Development of https://t.co/TD7xW96OHr
@Kevin_McKernan Yes it's ex-vivo but the methods will make you weep. They didn't even successfully test a clinical mouse model, only used surrogate markers. https://t.co/leCFVlaMgi
@EricTopol @NEJM Breakdown of the paper behind it: Title: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease This study describes the development and administration of a customized gene-editing therapy for a patient with a rare genetic disease called carbamoyl-phosphate https://t.co/5UpJsrbvcK
Personalized Gene Editing to Treat an Inborn Error of Metabolism www.nejm.org/doi/full/10.... Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease @ahrensnicklas.bsky.social @kiranmusunuru.bsky.social www.nejm.org/doi/full/10.... www.nejm.org/doi/full/10....
We are witnessing a historic revolution in medicine. For the first time, CRISPR gene editing has been successfully used to treat a baby with a previously incurable metabolic disease. This landmark treatment marks a…
A gene therapy innovation may have transformed medicine by saving a baby with a rare genetic disease. Following treatment with base-editing therapy via lipid nanoparticles, the baby showed significant improvement…
This video delves into the fundamental principles of quantum mechanics, explaining key concepts such as wave-particle duality and quantum entanglement. Viewers will gain insight into how these principles shape our…
A baby diagnosed with a rare genetic disorder received CRISPR gene editing directly in the liver. This innovative treatment, developed by a team from the University of Pennsylvania, has shown promising initial results…
A baby was cured of a rare disease after gene modification. This breakthrough involves precise CRISPR gene editing, highlighting recent advancements in personalized genetic treatments and their potential to save lives.
This video delves into the fundamentals of quantum mechanics, exploring its essential principles and theories. Learn about wave-particle duality, superposition, and entanglement, providing a foundational understanding…
A 9-month-old baby was saved using CRISPR—the revolutionary gene-editing technology. Born with CPS1 deficiency, the baby’s liver couldn't break down ammonia. After treatment with CRISPR and lipid nanoparticles, ammonia…
A baby treated with CRISPR for CPS1 deficiency shows significant improvement. This video explores the innovative gene-editing therapy developed to address severe genetic disorders, highlighting its impact and the…
Meet KJ Muldoon, born with a life-threatening genetic disorder. In February 2025, at just 7 months old, KJ received a groundbreaking personalized gene therapy. This video explores the promise of base editors in…
The development of individualized genetic therapies for rare diseases has a significant impact on affected patients and future treatment options. This video explores the promise of base editors in correcting harmful…
RT @EricTopol: Today "a milestone in the evolution of personalized therapies for rare & ultra-rare inborn errors of metabolism" —the 1st hu…
I came across a paper in NEJM on patient-specific in vivo gene editing for CPS1 deficiency. Groundbreaking because it shows personalized CRISPR-based therapy can save lives in rare genetic diseases. https://t.co/ImKHQBOEiC
Un bebé al que llamaron KJ, nacido con un trastorno genético que amenaza su vida, se convirtió en la primera persona en recibir un tratamiento personalizado de edición de genes CRISPR. ¿Es ético "diseñar genéticamente" a los hijos? DOI: 10.1056/NEJMoa2504747 Te contamos más de https://t.co/ctLR4gusWd
RT @NutrioSci: Personalized Gene Editing to Treat an Inborn Error of Metabolism #NEJM https://t.co/kIZmTPbRba https://t.co/5AQeUz3IW9 ht…
RT @NutrioSci: Personalized Gene Editing to Treat an Inborn Error of Metabolism #NEJM https://t.co/kIZmTPbRba https://t.co/5AQeUz3IW9 ht…
After a neonate had received a diagnosis of severe carbamoyl-phosphate synthetase 1 deficiency, a team of SCGE researchers developed and dosed the patient with a customized lipid nanoparticle-delivered base-editing therapy. #PublicationFriday https://t.co/z8xGlcCWZG
RT @NutrioSci: Personalized Gene Editing to Treat an Inborn Error of Metabolism #NEJM https://t.co/kIZmTPbRba https://t.co/5AQeUz3IW9 ht…
RT @NutrioSci: Personalized Gene Editing to Treat an Inborn Error of Metabolism #NEJM https://t.co/kIZmTPbRba https://t.co/5AQeUz3IW9 ht…
RT @NutrioSci: Personalized Gene Editing to Treat an Inborn Error of Metabolism #NEJM https://t.co/kIZmTPbRba https://t.co/5AQeUz3IW9 ht…
RT @NutrioSci: Personalized Gene Editing to Treat an Inborn Error of Metabolism #NEJM https://t.co/kIZmTPbRba https://t.co/5AQeUz3IW9 ht…
RT @NutrioSci: Personalized Gene Editing to Treat an Inborn Error of Metabolism #NEJM https://t.co/kIZmTPbRba https://t.co/5AQeUz3IW9 ht…
RT @NutrioSci: Personalized Gene Editing to Treat an Inborn Error of Metabolism #NEJM https://t.co/kIZmTPbRba https://t.co/5AQeUz3IW9 ht…
RT @NutrioSci: Personalized Gene Editing to Treat an Inborn Error of Metabolism #NEJM https://t.co/kIZmTPbRba https://t.co/5AQeUz3IW9 ht…
RT @NutrioSci: Personalized Gene Editing to Treat an Inborn Error of Metabolism #NEJM https://t.co/kIZmTPbRba https://t.co/5AQeUz3IW9 ht…
Personalized Gene Editing to Treat an Inborn Error of Metabolism #NEJM https://t.co/kIZmTPbRba https://t.co/5AQeUz3IW9 https://t.co/mHEjUJJNI8 https://t.co/5xy0dvAlyI
RT @jyasuda1: こちらが元論文。 https://t.co/j4fsjyOLJn
こちらが元論文。 https://t.co/j4fsjyOLJn
RT @EricTopol: Today "a milestone in the evolution of personalized therapies for rare & ultra-rare inborn errors of metabolism" —the 1st hu…
RT @gglinskii: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/MpUOLV…
RT @AIDrugsYRobots: https://t.co/mXkD7fjK7v
https://t.co/mXkD7fjK7v
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/MpUOLVOgbY
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/MpUOLVOgbY
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/MpUOLVNImq
Patient-specific in vivo gene editing to treat a rare genetic disease - Musunuru et al. 2025, NEJM https://t.co/75nl1a91vb https://t.co/Rmco4a9Wwu
RT @pnatarajanmd: This is a remarkable feat on so many levels. *Massive* congrats to @kiranmusunuru (former @Circ_Gen EiC & @skathire lab…
@NoVive_Socialis https://t.co/yzoURgEhSJ Gloriosa evolución diria el Viktor
@fakiardo_ https://t.co/e2BCQKdn7a Banco.
A fatal metabolic disease corrected in a six months old infant. "We anticipate that rapid deployment of patient-specific gene-editing therapies will become routine..." https://t.co/dSniTQQYaZ
At SeQure DX, we don’t just use cutting-edge assays — we invented them. ONE-seq + GUIDE-seq™ set the standard for off-target analysis, and helped power baby KJ’s lifesaving treatment. https://t.co/t0tFQkOwQx Let’s advance your program with confidence → https://t.co/O4Tvt7Px22 https://t.co/TCfiG3udak
Researchers diagnosed a baby with a life-threatening genetic disease days after birth, and spent 6 months designing & testing a personalized gene therapy to treat him. He could reduce meds, gain weight & move up on the growth chart. https://t.co/C8OaP7i6OR
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/HXe0zHz64G
RT @pnatarajanmd: This is a remarkable feat on so many levels. *Massive* congrats to @kiranmusunuru (former @Circ_Gen EiC & @skathire lab…
オーダーメイドのゲノム編集治療 米国の遺伝病赤ちゃんに世界初成功 5/16 https://t.co/uRDNmXk3u1 「全ゲノム解析」高まる期待、遅い取り組み 難病を診断、有効治療や創薬にも希望 6/29 https://t.co/pCj2J6Ik4Q NEJM https://t.co/Xi48OcAmXh Science News https://t.co/XFOm5Cd9Ba
RT @StevePascolo: Succès de l’ARNm synthétique: un bébé né avec 2 mutations (une dans le gène paternel et une dans le maternel) de CPS1 a r…
RT @StevePascolo: Succès de l’ARNm synthétique: un bébé né avec 2 mutations (une dans le gène paternel et une dans le maternel) de CPS1 a r…
RT @StevePascolo: Succès de l’ARNm synthétique: un bébé né avec 2 mutations (une dans le gène paternel et une dans le maternel) de CPS1 a r…
RT @StevePascolo: Succès de l’ARNm synthétique: un bébé né avec 2 mutations (une dans le gène paternel et une dans le maternel) de CPS1 a r…
RT @StevePascolo: Succès de l’ARNm synthétique: un bébé né avec 2 mutations (une dans le gène paternel et une dans le maternel) de CPS1 a r…
RT @StevePascolo: Succès de l’ARNm synthétique: un bébé né avec 2 mutations (une dans le gène paternel et une dans le maternel) de CPS1 a r…
RT @StevePascolo: Succès de l’ARNm synthétique: un bébé né avec 2 mutations (une dans le gène paternel et une dans le maternel) de CPS1 a r…
RT @StevePascolo: Succès de l’ARNm synthétique: un bébé né avec 2 mutations (une dans le gène paternel et une dans le maternel) de CPS1 a r…
Succès de l’ARNm synthétique: un bébé né avec 2 mutations (une dans le gène paternel et une dans le maternel) de CPS1 a reçu de l’ARNm codant un «éditeur» d’ADN et un ARN guide qui ont permis de «réparer» le gène dans les cellules du foie du bébé. https://t.co/ZM9iuwOxnT
https://t.co/TI6HD0HjIv
New England Journal of Medicine.
RT @NEJM: A lipid nanoparticle–delivered base-editing therapy was custom designed for an infant with a urea-cycle disorder. The affected in…
RT @NEJM: A lipid nanoparticle–delivered base-editing therapy was custom designed for an infant with a urea-cycle disorder. The affected in…
RT @NEJM: A lipid nanoparticle–delivered base-editing therapy was custom designed for an infant with a urea-cycle disorder. The affected in…
A lipid nanoparticle–delivered base-editing therapy was custom designed for an infant with a urea-cycle disorder. The affected infant was treated at approximately 7 and 8 months of age. Read the Brief Report: https://t.co/EtnnBhGfqi Editorial: Progress in the Development of https://t.co/TD7xW96OHr
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/KY6WPgY4OD
@Bonemaxxer @sense_strand Literally linked in my quote tweet. Not off to a great start are you? https://t.co/isFwu2B7Up https://t.co/IHYzyN0PEo
Patient-Specific #In Vivo Gene Editing to Treat a #Rare Genetic Disease https://t.co/zJhDimBjkZ
RT @EricTopol: Today "a milestone in the evolution of personalized therapies for rare & ultra-rare inborn errors of metabolism" —the 1st hu…
@ElAnnimo781868 @xixio3d @abc_es Sí hubo fases de ensayos clínicos, además de ser una tecnología estudiada a lo largo de muchos años, la razón por la que salió rápido es porque gobiernos y científicos de todo el mundo le dieron prioridad, mismo caso que esté niño: https://t.co/yM5taZi5cy
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/6lrsdLkkvy #RareDisease #Genetics #GeneEditing
Edición genética in vivo específica en un neonato con deficiencia grave de carbamoilfosfato sintetasa 1, de mortalidad del 50%, comenzó terapia de edición de bases personalizada mediante nanopartículas lipídicas. https://t.co/J5Aa6CxysX
Reported in @NEJM today: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/7oiHZ4QSVD
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/wF46wdHi8V
https://t.co/Fn2UtW8uoJ
RT @Jikkyleaks: @Kevin_McKernan Yes it's ex-vivo but the methods will make you weep. They didn't even successfully test a clinical mouse…
RT @Jikkyleaks: @Kevin_McKernan Yes it's ex-vivo but the methods will make you weep. They didn't even successfully test a clinical mouse…
RT @Jikkyleaks: @Kevin_McKernan Yes it's ex-vivo but the methods will make you weep. They didn't even successfully test a clinical mouse…
RT @Jikkyleaks: @Kevin_McKernan Yes it's ex-vivo but the methods will make you weep. They didn't even successfully test a clinical mouse…
RT @Jikkyleaks: @Kevin_McKernan Yes it's ex-vivo but the methods will make you weep. They didn't even successfully test a clinical mouse…
RT @Jikkyleaks: @Kevin_McKernan Yes it's ex-vivo but the methods will make you weep. They didn't even successfully test a clinical mouse…
@Kevin_McKernan Yes it's ex-vivo but the methods will make you weep. They didn't even successfully test a clinical mouse model, only used surrogate markers. https://t.co/leCFVlaMgi
RT @DOGE_VIROLOGY:
RT @Navaraaa: @Vaalxeny https://t.co/6LEyTOS19M
@Vaalxeny https://t.co/6LEyTOS19M
They will discuss implications of their research on using patient-specific in vivo gene editing to treat a rare genetic disease. Register to hear about the historic medical breakthrough during this year’s impressive two-week speaker lineup: www.jax.org/education-an... #GeneTherapy #RareDisease
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/XKidbwH6S2
Acabo de ver un vídeo verdaderamente increíble. @lahiperactina explica lo del bebé que curaron hace poco con #CRISPR, todo un hito en #Biomedicina. Por estas cosas apoyo al #Capitalismo, porque salva vidas. https://t.co/0EWMn6NHxm
Co-first authorship for Sarah Grandinette in the @kiranmusunuru.bsky.social lab on: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine (@NEJM.org) www.nejm.org/doi/full/10....
▶️ Se aplicó una edición genética in vivo personalizada para tratar una enfermedad genética rara https://t.co/oYRCfdfKsM https://t.co/hEvzOA5NPH
▶️ Descubrieron un nuevo blanco terapéutico para frenar tumores intracraneales https://t.co/oYRCfdfKsM https://t.co/renqbxmG1k
RT @CAMBUpenn: Co-first authorship for Sarah Grandinette (@Sarah_Grand) in the @kiranmusunuru lab on: Patient-Specific In Vivo Gene Editing…
Co-first authorship for Sarah Grandinette (@Sarah_Grand) in the @kiranmusunuru lab on: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine (@NEJM) https://t.co/LXNaHOZRa2
Patient-Specific In Vivo Gene Editing to Treat a Rare #Genetic Disease | New England Journal of Medicine https://t.co/aLFS9VoxUN #RareDiseases A great promise @Gyan_Lab @bioclues
RT @pnatarajanmd: This is a remarkable feat on so many levels. *Massive* congrats to @kiranmusunuru (former @Circ_Gen EiC & @skathire lab…
RT @SintoniaSecreta: Una gran noticia. Aquest és el camí https://t.co/d4WXFKHvwC
¡#CRISPR FUNCIONA! PRIMER BEBÉ curado con EDICIÓN #GENÉTICA
#PaginaGialla: raro, ultrararo, strepitoso: N-of-1 ovvero editing genetico 2.0 su NEJM Neonato con forma gravissima di deficit di carbamilfosfato sintetasi 1 trattato con editing genetico su misura per la sua specifica mutazione https://t.co/iukgQJkvsM https://t.co/pc6eDKCif4
https://t.co/q6EkyGznkJ
Esto debería ser noticia, el mundo está apunto de cambiar. https://t.co/1BiEnhGzXC
RT @SintoniaSecreta: Una gran noticia. Aquest és el camí https://t.co/d4WXFKHvwC
RT @SintoniaSecreta: Una gran noticia. Aquest és el camí https://t.co/d4WXFKHvwC
Una gran noticia. Aquest és el camí https://t.co/d4WXFKHvwC
9/
Con buena voluntad .... https://t.co/QgjWsSeALv
Curan a un bebé con CRISPR por primera vez https://www.youtube.com/watch?v=JjFNjIS7xlc
Curan a un bebé con CRISPR por primera vez https://t.co/DJeMsTYXBI
RT @EricTopol: Today "a milestone in the evolution of personalized therapies for rare & ultra-rare inborn errors of metabolism" —the 1st hu…
CURAN a un BEBÉ con CRISPR por PRIMERA VEZ https://t.co/lAXRf6llEj a través de @YouTube
Y por acabar con una noticia positiva, un hito en medicina: https://t.co/63SLx3hEjX #Medicina #CRISPR #NoticiasOptimistas
Se usó edición genética (CRISPR) para modificar el ADN de un bebé y curarlo de una enfermedad genética hasta ese momento incurable https://t.co/tRFz2WD6pA
RT @EricTopol: Today "a milestone in the evolution of personalized therapies for rare & ultra-rare inborn errors of metabolism" —the 1st hu…
Dr. Kiran Musunuru, @Sarah_Grand, Dr. Rebecca @AhrensNicklas, and a team at @PennMedicine used #GeneEditing to treat severe carbamoyl-phosphate synthetase 1 deficiency, a rare genetic disease!
RT @pnatarajanmd: This is a remarkable feat on so many levels. *Massive* congrats to @kiranmusunuru (former @Circ_Gen EiC & @skathire lab…
Per la prima volta al mondo, un neonato ha ricevuto una terapia #CRISPR su misura per correggere una mutazione genetica rara. Il trattamento è stato sviluppato in soli 6 mesi. https://t.co/7Mkt3QKwwV https://t.co/GXw7gdEzzs
RT @pnatarajanmd: This is a remarkable feat on so many levels. *Massive* congrats to @kiranmusunuru (former @Circ_Gen EiC & @skathire lab…
Lo que hablaba @tomasrebord en la última #edibordial https://t.co/MDx2ceRYLm
CURAN a un BEBÉ con CRISPR por PRIMERA VEZ https://t.co/h44RxhLub0 a través de @YouTube
Los avances en la investigación en genética y medicina permiten esta buena noticia: CURAN a un BEBÉ con CRISPR por PRIMERA VEZ https://t.co/skHIu3fBB9 a través de @YouTube
CURAN a un BEBÉ con CRISPR por PRIMERA VEZ https://t.co/YtbcVUTaIE a través de @YouTube
@eigenrobot They devote their whole lives to curing the sick and achieve things like this. Your hostility is mystifying and unwarranted. https://t.co/VIgtRbH5mR
RT @erlesen: Patient-Specific in vivo gene editing to treat carbamoyl-phosphate synthetase 1 deficiency https://t.co/KaTZ3LK4zd Researchers…
@svetloplava13 Musunuru et al, “Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease.” N Engl J Med. Online May 15, 2025. DOI: 10.1056/NEJMoa25
#apaperaday is back from holiday. Yuzu is still with the birbsitter so a holiday picture it is. Today's pick is from @nejm.org by Musunuru et al on an N=1 case (patient with severe metabolic disease) treated by base editing (CRISPR therapy). DOI: 10.1056/NEJMoa2504747 Very good way to restart!
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine @NEJM if you sign up, they will send you the article, but they won’t. They’ll demand you subscribe; Liars, and thieves. https://t.co/XruBJQjvbl
https://t.co/HwqxDhYdSw
@Amirali_tk مقاله اصلی رو نیچره واسه خوندش فکر کنم مجانی نباشی https://t.co/dGcXpkNlqA
@a27040669 https://t.co/dGcXpkNlqA
¿Quieres saber más sobre este fascinante caso y la ciencia detrás de #CRISPR personalizado? Explora estas fuentes originales: Nature: https://t.co/v9DJ6w4y2L IGI: https://t.co/z75ANYQPLV NEJM:https://t.co/bFrvvWHJ2u NYT:https://t.co/wuAG7v1y8K #Ciencia #DivulgaciónCientífica
RT @EricTopol: Today "a milestone in the evolution of personalized therapies for rare & ultra-rare inborn errors of metabolism" —the 1st hu…
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. https://t.co/BqEY2xbPGD
RT @EricTopol: Today "a milestone in the evolution of personalized therapies for rare & ultra-rare inborn errors of metabolism" —the 1st hu…
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. https://t.co/BqEY2xbPGD
RT @pnatarajanmd: This is a remarkable feat on so many levels. *Massive* congrats to @kiranmusunuru (former @Circ_Gen EiC & @skathire lab…
RT @BlueJakk: به خواست #خدا این کوچولو با کبدی متولد شده بود که نمیتونسته آمونیاک را به اوره تبدیل کنه اما طبق معمول عزیزان از طراحی الهی خ…
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/URvOGXfp6p
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/Xna9wnOR4B
RT @FilippoMontemu1: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/b…
RT @EricTopol: Today "a milestone in the evolution of personalized therapies for rare & ultra-rare inborn errors of metabolism" —the 1st hu…
RT @BlueJakk: به خواست #خدا این کوچولو با کبدی متولد شده بود که نمیتونسته آمونیاک را به اوره تبدیل کنه اما طبق معمول عزیزان از طراحی الهی خ…
RT @BlueJakk: به خواست #خدا این کوچولو با کبدی متولد شده بود که نمیتونسته آمونیاک را به اوره تبدیل کنه اما طبق معمول عزیزان از طراحی الهی خ…
RT @broadinstitute: We’re thrilled that base editing, developed by researchers in the lab of Broad member @davidrliu, is having such a prof…
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. https://t.co/BqEY2xbPGD
@dago74173451 już robią - w Chinach (na zarodkach) całe lata temu, u nas tzn. w USA na 7 tyg. dziecku - teraz. https://t.co/IWOkGbqkCS
RT @BlueJakk: به خواست #خدا این کوچولو با کبدی متولد شده بود که نمیتونسته آمونیاک را به اوره تبدیل کنه اما طبق معمول عزیزان از طراحی الهی خ…
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/JFi2qan3xn
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/bKGHeWcMdB The astonishing thing in this single subject study, is the workflow that led to delivering of personalized base editing in a rare genetic diseas.
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/A1hbotZCW7
Adoro divulgador científico, essa mistura de pesquisador, professor e comunicador. Parabéns aos pioneiros dessa nova profissão. O Brasil precisa reconhecer a profissão de cientista e de divulgador científico @paulopaim https://t.co/d1C3rfRsW9
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. https://t.co/BqEY2xbPGD
https://t.co/JdRi452B5S
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. https://t.co/BqEY2xbPGD
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. https://t.co/BqEY2xbPGD
Exciting breakthrough! A baby with a severe genetic disorder received a tailored gene-editing therapy, allowing them to eat more protein and reduce medication, all without major side effects. More research needed! - Read more: https://t.co/ynr2sQHEUI
RT @chakrabartyd: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/oepN…
カルバモイルリン酸合成酵素1欠損症の患児に遺伝子編集治療実施、高アンモニア血症改善 Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/Jt8yF742GF
Patient-Specific In Vivo #Gene Editing to Treat a Rare Genetic Disease https://t.co/zJhDimBjkZ
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/oepNg5PWlb
RT @BensenHsu: @EricTopol Breakdown of the paper behind it: Title: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease…
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. https://t.co/BqEY2xbPGD
Los resultados fueron prometedores para otras personas con afecciones genéticas, afirmó el equipo médico, que publicó su estudio el jueves en el New England Journal of Medicine (https://t.co/CAJjTmidZM)
RT @NatMed_Research:
RT @NatMed_Research:
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Breakdown of the paper behind it: Title: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease This study focuses on a very rare genetic disorder called carbamoyl-phosphate synthetase 1 deficiency, which affects how the body processes waste products from protein...
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RT @BensenHsu: @EricTopol Breakdown of the paper behind it: Title: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease…
@EricTopol Breakdown of the paper behind it: Title: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease This study focuses on a very rare genetic disorder called carbamoyl-phosphate synthetase 1 deficiency, which affects how the body processes waste products from https://t.co/46YOhnD9x8
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RT @AtualizadoSaber: (2) Publicação do estudo: Musunuru et al. (2025). Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disea…
RT @NEJM: Brief Report: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/EtnnBhGfqi Editorial: Progress…
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/cMrUkfFvdl
RT @SaraGutierrezEn: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/S…
RT @Satyayadav__: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease Baby was treated by in vivo base editing https://…
RT @NEJM: Brief Report: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/EtnnBhGfqi Editorial: Progress…
RT @AbdulAlgarni: Breakthrough! A new study demonstrates in vivo gene editing tailored to individual patients, offering hope for rare genet…
Breakthrough! A new study demonstrates in vivo gene editing tailored to individual patients, offering hope for rare genetic diseases. Precision medicine takes a giant leap forward https://t.co/amwYL3DJwW
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. https://t.co/BqEY2xbPGD
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/07ABys3ghn
RT @NEJM: Brief Report: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/EtnnBhGfqi Editorial: Progress…
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/0ha9b8g42C
RT @SaraGutierrezEn: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/S…
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease #science #publication #research #journal https://t.co/EpUT5lOaco
RT @SaraGutierrezEn: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/S…
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/SNQ9zPKweQ
Um marco para a ciência: a primeira edição genética para corrigir um defeito genético em um recém nascido! Que o futuro se materialize! Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/Mhysfk2q3u
RT @NEJM: Brief Report: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/EtnnBhGfqi Editorial: Progress…
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/z9nZhPXpGb
RT @NEJM: Brief Report: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/EtnnBhGfqi Editorial: Progress…
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine www.nejm.org/doi/10.1056/...
RT @BioEdge21: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/kCmkd5L…
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(2) Publicação do estudo: Musunuru et al. (2025). Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. NEJM. doi.org/10.1056/NEJM... Referência adicional: www.nature.com/articles/d41...
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/4e9Rd2jxge
(2) Publicação do estudo: Musunuru et al. (2025). Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. NEJM. https://t.co/LkphVeWPYj Referência adicional: https://t.co/K1LYbu4Oeh
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease www.nejm.org/doi/full/10.... #crispr #geneediting #precisionmedicine
RT @MoradiShayan: First in-human in vivo gene editing success: beautiful work of @kiranmusunuru and the team reports base editing used to t…
RT @AgingBiology: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/hItz61dykM
DOI: 10.1056/NEJMoa2504747
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine. Congrats CHOP! https://t.co/u169TrHLhA
RT @zoewangai: @EricTopol @NEJM Breakdown of the paper behind it: Title: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Dis…
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/kCmkd5LXoG
RT @NEJM: Brief Report: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/EtnnBhGfqi Editorial: Progress…
The paper in "NEJM" the above "Nature" article is based upon, & links to: “Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease” 2/ https://t.co/7fKeYQwJca
RT @MoradiShayan: First in-human in vivo gene editing success: beautiful work of @kiranmusunuru and the team reports base editing used to t…
RT @NEJM: Brief Report: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/EtnnBhGfqi Editorial: Progress…
RT @MoradiShayan: First in-human in vivo gene editing success: beautiful work of @kiranmusunuru and the team reports base editing used to t…
RT @NEJM: Brief Report: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/EtnnBhGfqi Editorial: Progress…
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine www.nejm.org/doi/full/10....
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/hItz61dykM
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease www.nejm.org/doi/full/10....
RT @NEJM: Brief Report: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/EtnnBhGfqi Editorial: Progress…
RT @NEJM: Brief Report: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/EtnnBhGfqi Editorial: Progress…
RT @NEJM: Brief Report: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/EtnnBhGfqi Editorial: Progress…
RT @NEJM: Brief Report: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/EtnnBhGfqi Editorial: Progress…
Brief Report: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/EtnnBhGfqi Editorial: Progress in the Development of N-of-1 Therapy https://t.co/YTdlxTQ7oN Science behind the Study: Personalized Gene Editing to Treat an Inborn Error of https://t.co/Vc2po1n8aB
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/hx8lQX3MA6 It is amazing to see how CRISPR has been used in an infant to correct a genetic disease due to a base mutation. Basic research is so essential!
RT @zoewangai: @EricTopol @NEJM Breakdown of the paper behind it: Title: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Dis…
What an incredible day with work from the Children Hospital of Philadelphia, @pennmedicine.bsky.social and their colleagues! First-ever personalized #LNP -based #CRISPR #GeneTherapy for the rare genetic condition carbamoyl-phosphate synthetase 1 ( #CPS1) deficiency. www.nejm.org/doi/full/10....
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. https://t.co/BqEY2xbPGD
Progress is based on Science. Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine www.nejm.org/doi/full/10....
RT @Satyayadav__: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease Baby was treated by in vivo base editing https://…
RT @zoewangai: @EricTopol @NEJM Breakdown of the paper behind it: Title: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Dis…
RT @MoradiShayan: First in-human in vivo gene editing success: beautiful work of @kiranmusunuru and the team reports base editing used to t…
RT @MoradiShayan: First in-human in vivo gene editing success: beautiful work of @kiranmusunuru and the team reports base editing used to t…
Wow! Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | NEJM www.nejm.org/doi/full/10....
First in-human in vivo gene editing success: beautiful work of @kiranmusunuru and the team reports base editing used to treat a newborn with CPS1 deficiency—ammonia levels dropped, no adverse effects. A milestone for personalized genomic medicine. https://t.co/WNDhPNxR7U @NEJM
Patient-Specific in vivo gene editing to treat carbamoyl-phosphate synthetase 1 deficiency www.nejm.org/doi/full/10.... Researchers in the USA have successfully treated a child with personalized gene therapy within a few months for the first time – a milestone! www.tagesschau.de/wissen/gesun...
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of https://t.co/Hcgsgn23te, Landmark moment: doctors used customized gene editing inside a living baby to treat a deadly genetic disease called carbamoyl-phosphate synthetase 1 (CPS1) https://t.co/TmFm5EHO9i
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of https://t.co/Hcgsgn23te, Landmark moment: doctors used customized gene editing inside a living baby to treat a deadly genetic disease called carbamoyl-phosphate synthetase 1 (CPS1)
RT @zoewangai: @EricTopol @NEJM Breakdown of the paper behind it: Title: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Dis…
RT @ScienceCrisp: "Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease" Musunuru K [..] Urnov FD, Ahrens-Nicklas RC. N…
CRISPR-Cas9による個別化遺伝子編集治療！すごい時代がきました。 Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/7qd6oohLh3
RT @zoewangai: @EricTopol @NEJM Breakdown of the paper behind it: Title: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Dis…
RT @weldeiry: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/x8WOIr1z97
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine | @scoopit https://t.co/E3N0p3S5UF
RT @Satyayadav__: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease Baby was treated by in vivo base editing https://…
RT @zoewangai: @EricTopol @NEJM Breakdown of the paper behind it: Title: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Dis…
RT @KSusztak: #Whyscience day41 Congrats to @kiranmusunuru and Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | @nej…
RT @zoewangai: @EricTopol @NEJM Breakdown of the paper behind it: Title: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Dis…
RT @KSusztak: #Whyscience day41 Congrats to @kiranmusunuru and Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | @nej…
希少遺伝性疾患のカルバミルリン酸合成酵素1欠損症の乳児が、CRISPR遺伝子編集を使ったカスタマイズ治療を受け、良好な反応を示した。生殖細胞以外の細胞を標的とするように設計されているので、変化は患者自身にのみ影響を及ぼす。New England Journal of Medicine誌。 https://t.co/8Swe7qfM11
RT @LabWaggoner: Personalized Gene Editing to Treat an Inborn Error of Metabolism https://t.co/D5QyzeAcv5 Patient-Specific In Vivo Gene Edi…
RT @zoewangai: @EricTopol @NEJM Breakdown of the paper behind it: Title: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Dis…
@EricTopol @NEJM Breakdown of the paper behind it: Title: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease This study describes the development and administration of a customized gene-editing therapy for a patient with a rare genetic disease called carbamoyl-phosphate https://t.co/5UpJsrbvcK
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease www.nejm.org/doi/pdf/10.1...
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://t.co/x8WOIr1z97
Personalized Gene Editing to Treat an Inborn Error of Metabolism https://t.co/D5QyzeAcv5 Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease @AhrensNicklas @kiranmusunuru https://t.co/L4dcPddy2n https://t.co/WDGt92VXSZ https://t.co/esBSrXR5tk
Personalized Gene Editing to Treat an Inborn Error of Metabolism www.nejm.org/doi/full/10.... Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease @ahrensnicklas.bsky.social @kiranmusunuru.bsky.social www.nejm.org/doi/full/10.... www.nejm.org/doi/full/10....
"Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease" Musunuru K [..] Urnov FD, Ahrens-Nicklas RC. N Engl J Med 2025-05-15 https://t.co/SMQWo6454R Carbamoyl-Phosphate Synthetase 1 deficiency. #CRISPR k-abe: NGC-ABE8e-V106W targeting CPS1<Q335X>) / LNP
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease Baby was treated by in vivo base editing https://t.co/SiuCOcd4dG
Wow this incredible. Using CRISPR in a human patient successfully. Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine www.nejm.org/doi/full/10....
Terapia Personalizada en Enfermedad rara!!!! CRISP-R https://t.co/Hl8r2oXjpQ via @YouTube
RT @BowTiedBiotech: And will add one more from D.Liu - incredible. Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease…
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine https://t.co/YXup7Y4cOI
https://www.theguardian.com/science/2025/may/15/us-doctors-rewrite-dna-of-infant-with-severe-genetic-disorder-in-medical-first; https://www.nejm.org/doi/full/10.1056/NEJMoa2504747 (abstract only). Before we all shout "Hoorah!", let's consider the ethical implications, shall we, @Peternimmo? This […]
Incredible research published in the New England Journal of Medicine today: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine www.nejm.org/doi/full/10.... Congratulations to Tri-I Alums @kiranmusunuru.bsky.social and @ahrensnicklas.bsky.social!
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine www.nejm.org/doi/full/10....
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease https://lobste.rs/s/moznqb #science
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine www.nejm.org/doi/full/10....
And original article here: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | New England Journal of Medicine www.nejm.org/doi/full/10....