The collection broadly treats the NEJM paper as a milestone in CRISPR-based therapy for SCD and β-thalassemia. Posts frequently highlight the mechanism (downregulating BCL11A to boost HbF), the ex vivo stem-cell approach, and initial patient benefits. Tone ranges from celebratory and promotional to cautious or informational. Word choices like breakthrough, milestone, transfusion independence, and potential to cure shape an overall narrative of excitement and cautious optimism about real-world impact, while occasional critical notes reference ongoing research, safety, and the need for extended follow-up.
AgreementModerate agreement
Overall positive alignment with the article, many posts praise the breakthrough and express approval, though a few skeptical or contextual caveats exist.
InterestHigh interest
High intrinsic interest across posts, with many references to HbF induction, BCL11A enhancer, and clinical implications.
EngagementModerate engagement
Moderate to high engagement: some posts summarize details, share sources, or advocate reading, while others are simply reposts or brief mentions.
ImpactHigh impact
Very high potential impact discussed: transfusion independence, reduced vaso-occlusive events, and broader gene-editing implications.
Abstract in brief
Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are serious genetic disorders that require ongoing medical treatment and can be life-threatening.
Researchers utilized CRISPR-Cas9 technology to modify CD34+ stem cells from healthy donors, targeting a specific enhancer to increase fetal hemoglobin production by altering BCL11A, a gene that suppresses it.
Two patients, one with TDT and the other with SCD, received these edited cells after a preparative procedure and showed significant improvements after a year, including high fetal hemoglobin levels, independence from blood transfusions, and reduced complications from SCD.
And where is the edit made?
In an enhancer region for the BCL11A gene.
Turning this gene off increases the level of fetal hemoglobin
https://t.co/kF6NWC5UsC https://t.co/ESGN8I48GZ
Jun 11, 2022 · 45 likes · 5 shares · 30,190 followersView post →
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Stefano Berto, PhD
@StefanoBerto83 · X
Ad oggi ci sono due terapie approvate: una per l'anemia falciforme e l'altra per la Beta talassemia
In questo caso CRISPR è stato usato per correggere le
cellule staminali emopoietiche del paziente eliminando la funzione repressiva di BCL11A
https://t.co/GwUpqU6hBv
19/
Dec 11, 2024 · 27 likes · 7,880 followersView post →
SG
Sebastián Giusti
@SebasGiusti · X
Another 2020 Scientific Breakthrough: CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia | NEJM https://t.co/wrS9RXnkyg
Dec 20, 2020 · 27 likes · 2 shares · 2,505 followersView post →
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Scienceyoushouldknow
@ScienceYSK · X
Dopo aver visto due approcci B1, spostiamoci verso un approccio A'2 (modifica al genoma + cellule ematopoietiche staminali ex vivo). Ad esempio, ecco altri dati clinici presentati alla Nature Conference da Haydar Frangoul. Finalmente parlo di CRISPR.
https://t.co/qjRJYXyynQ 10/19
Nov 21, 2021 · 24 likes · 3,452 followersView post →
IF
Ishwaria Subbiah, MD MS FASCO
@IshwariaMD · X
Dec 9, 2023 · 16 likes · 3 shares · 9,493 followersView post →
峰
峰 宗太郎
@minesoh · X
NEJM
Dec 8, 2020 · 14 likes · 1 share · 70,836 followersView post →
Researchers used CRISPR-Cas9 to modify stem cells, increasing fetal hemoglobin to treat serious genetic disorders like TDT and SCD, showing promising results in patients after one year.
A four-month randomized trial tested Urolithin A, a postbiotic that promotes mitophagy, for middle-aged adults. Participants showed improved muscle strength, aerobic endurance, and mitochondrial efficiency, with reduced…
This video discusses the recent use of CRISPR-Cas9 technology to modify stem cells for treating genetic blood disorders. It highlights significant improvements in patients with TDT and SCD, including increased fetal…
Ad oggi ci sono due terapie approvate: una per l'anemia falciforme e l'altra per la Beta talassemia
In questo caso CRISPR è stato usato per correggere le
cellule staminali emopoietiche del paziente eliminando la funzione repressiva di BCL11A
https://t.co/GwUpqU6hBv
19/
Dec 11, 2024 · 27 likes · 7,880 followersView post →
DM
Dr Jérôme BARRIÈRE - MD
@drbarriere.bsky.social · Bluesky
?réduction significative des crises douloureuses chez les patients drépanocytaires
?diminution ou une élimination des besoins en transfusions chez les patients atteints de β-thalassémie.
www.nejm.org/doi/full/10....
7/⤵️
The ability to cut out DNA mutations (thanks Darwin), replacing them with non-mutated segments, promises within the next few years to allow the eradication of a small number of purely genetic diseases such as sickle cell anaemia without further medicine: www.altmetric.com/details/9549...
16/x
Dec 10, 2023 · 3 shares · 333 followersView post →
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Sahba Ferdowsi DO (conciergedoc)
@DrFerdowsi · X
RT @IshwariaMD:
Dec 10, 2023 · 3 shares · 9,633 followersView post →
VM
Vivek Subbiah, MD
@VivekSubbiah · X
RT @IshwariaMD:
Dec 9, 2023 · 3 shares · 39,526 followersView post →
IF
Ishwaria Subbiah, MD MS FASCO
@IshwariaMD · X
Dec 9, 2023 · 16 likes · 3 shares · 9,493 followersView post →
KM
Kiran Musunuru
@kiranmusunuru · X
@skathire @GLettre @bloodgenes @joelhirschhorn @JianXuLab @danielevanbauer 9/
$VRTX and $CRSP developed a CRISPR therapy—exa-cel—that edits the BCL11A erythroid enhancer in bone marrow stem cells of patients with SCD or beta thalassemia (another blood disorder in which increased HbF is beneficial).
https://t.co/f5Vggs5jYA
RT @HealthMotifs: Casgevy is a trademark name for a novel gene therapy that uses CRISPR-Cas9 gene editing for sickle cell disease and β-tha…
Nov 29, 2023 · 1 share · 6,749 followersView post →
HM
Health Motifs
@HealthMotifs · X
Casgevy is a trademark name for a novel gene therapy that uses CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia, two inherited blood disorders...Keep Reading at
https://t.co/NBpwBUQWl1
#CRISPR #UK #IsraelisGenocidalState
#crispr #sickel #thalasemia
The MHRA approved Casgevy to treat sickle-cell disease and transfusion-dependent beta-thalassemia. These are genetic disorders caused by mutations in the hemoglobin gene, a protein that red blood cells need to transport oxygen
https://t.co/Heapit7szM
Nov 24, 2023 · 2 likes · 1 share · 1,337 followersView post →
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SiyuanTao
@SiyuanTao_cn · X
CRISPR-Cas9 gene therapy CTX001 shows potential in treating certain blood disorders, with positive early results indicating a need for extended research. #CRISPR #HealthInnovation
https://t.co/w6pxgLrKmt
https://t.co/Xb7QnDNynE
"SCD denotes sickle cell disease, and TDT transfusion-dependent β-thalassemia. Panel B shows the target editing site of the single guide RNA ( ..."
@VIXTiming @IdoWolf1 המחקר הנוכחי עוסק ב-Sickle Cell Disease בלבד
אבל המחקר הזה
Aug 31, 2023 · 1 like · 3,110 followersView post →
SD
Strange Demon
@DeleuzeMD · X
@bayesianboy High turnover cells without anatomic constraints have already been targeted (eg https://t.co/viVqxKE9fj
https://t.co/w4Jb6uV6RK)
Neural transcription modulation is emerging but doesn’t affect the germline, only the RNA expression (https://t.co/ciSKAQOWrE)
@AsherAharonov @StavKislev והאדם יכול לתפקד בצורה תקינה ביומיום עם העמידות למלריה רק אם יש גם עותק של אלל תקין ליצירת תאי דם.
אם אין, צריך המון עירויי דם בשנה. מה שמעניין, יש כבר בכמה ניסויים קליניים טיפוליים מבוססי קריספר לריפוי (במידה מסוימת) של אנמיה חרמשית ובטא תלסמיה.
https://t.co/eTzZrURkso
Jun 12, 2023 · 2 likes · 1,763 followersView post →
May 24, 2023 · 2 likes · 3,004 followersView post →
MI
Medic info
@KonczalikWojtek · X
#CRISPR #Thalassemia #hematology
There is huge interest in the use of CRISPR/Cas9-mediated beta-globin (HBB) gene correction of sickle cell disease (SCD) and thalasemmia - this could be a game changer for millions of patients globally
@magichourtrave1 @zap_lock I'm not aware of the widespread use of
"experimental gene therapy jabs".
Are you referring to use of CRISPR-Cas9 ?
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
https://t.co/plKJCGXx7Z
I'm tracking that for personal reasons as a blood donor: https://t.co/rVMKchw3JB
Lots of subsequent fundamental and pre-clinical studies have led to recent clinical trials that suppress BCL11A for induction of HbF by gene therapy/ editing approaches:
https://t.co/q99VShoFQE
https://t.co/uCpplX9V4j
(4/n)
Mar 24, 2023 · 6 likes · 11,572 followersView post →
P
peecheegeechee
@peecheegeechee · X
@nching0 This article from the New England journal of medicine shows that trial participants are 18-35. https://t.co/8w7GhbB2y6
RT @LluisMontoliu: The science behind Victoria Gray’s #CRISPR sickle cell disease treatment was described in this article by Frangoul et al…
Mar 6, 2023 · 4 shares · 47,742 followersView post →
LM
Lluis Montoliu
@LluisMontoliu · X
The science behind Victoria Gray’s #CRISPR sickle cell disease treatment was described in this article by Frangoul et al. published in @NEJM
#HGESummit
https://t.co/cfDs8vk2FH
Mar 6, 2023 · 9 likes · 4 shares · 38,440 followersView post →
DG
Dr. Antonio Gutiérrez
@boticariors · X
Datos preliminares sobre uso de herramienta #CRISPR en Sickle Cell Disease and β-Thalassemia
https://t.co/H7iPWtzR5j
A recent study present by Haydar Frangoul team shows a medical treatment based on CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia.
This is the first approved medical treatment by CRISPR-Cas9!
https://t.co/968nYB4Gwk
The first set of clinical trials was published in 2021. One in sickle cell anemia and the other in beta thalassemia. There is an informative short video in the link explaining both studies.
1.https://t.co/cmJBPLPA6b
2.https://t.co/FFVstJ018Z
Clinical trial saying everyone was cured (oh by the way...):
https://t.co/bfByajC3Ub
Shows that CRISPRs can be a therapy in these diseases:
https://t.co/b2duzoSyMw
Lays groundwork for putzing with BCL11A:
https://t.co/krouKQD03a
And where is the edit made?
In an enhancer region for the BCL11A gene.
Turning this gene off increases the level of fetal hemoglobin
https://t.co/kF6NWC5UsC https://t.co/ESGN8I48GZ
Jun 11, 2022 · 45 likes · 5 shares · 30,190 followersView post →
C
clunky!
@Clunkyyy · X
@TroyRiser @Loveeve65702806 @MetroUK Think good outcomes & reconsider.
https://t.co/u4gDvLDHSC
@Di_Lucca68 @GabiTCC @ibrahimkadis @GVDBossche In real gene therapy, you see the effects in the first month. You need a vehicle (viral envelope) to deliver the gene. mRNA doesn’t just easily integrate. Why would you be scared of a few doses of mRNA by itself and not mRNA from a replicating virus? https://t.co/cl7h9wq0Q8
Dopo aver visto due approcci B1, spostiamoci verso un approccio A'2 (modifica al genoma + cellule ematopoietiche staminali ex vivo). Ad esempio, ecco altri dati clinici presentati alla Nature Conference da Haydar Frangoul. Finalmente parlo di CRISPR.
https://t.co/qjRJYXyynQ 10/19
Nov 21, 2021 · 24 likes · 3,452 followersView post →
IS
Isatou Sarr
@IsatouSarr4 · X
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia https://t.co/oZg5kIPEvu
RT @alexmd2: The protocol is tough but the results are worth the risk.
https://t.co/of8IbZSKV2
Oct 30, 2021 · 1 share · 1,645 followersView post →
TS
Tilmann Schober
@TilmannSchober · X
@MarisaKurz @sabine_av @johannesboie @lmu @BILD Anderes Beispiel: CRISPR Studie zu SCD, andere Forscher, aber auch aus D, auch im NEJM publiziert, auch absolutes Neuland. Wir haben bislang kein Follow-up? Lehnen Sie die Arbeit auch ab?
P.S. Ich finde die Arbeit grossartig!
https://t.co/WPYcQ6k0fi
RT @Pathologists: CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. Register for this CAP Personalized Healthcare Committ…
Oct 12, 2021 · 2 shares · 1,516 followersView post →
C
CAPathologists
@Pathologists · X
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. Register for this CAP Personalized Healthcare Committee 10/19 webinar featuring Haydar Frangoul, MD, MS, FCAP: https://t.co/gIoekEGvba. https://t.co/6H98kS5MHx
Oct 12, 2021 · 2 likes · 2 shares · 32,876 followersView post →
دا
د.محمد مشبب القحطاني
@godmah86 · X
RT @Pathologists: CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. Register for this CAP Personalized Healthcare Committ…
Oct 8, 2021 · 1 share · 21,532 followersView post →
C
CAPathologists
@Pathologists · X
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. Register for this CAP Personalized Healthcare Committee 10/19 webinar featuring Haydar Frangoul, MD, MS, FCAP: https://t.co/gIoekEGvba. https://t.co/c97blVsddP
Oct 8, 2021 · 3 likes · 1 share · 32,876 followersView post →
FU
Fyodor Urnov
@UrnovFyodor · X
@PetrovADmitri @ambikamath Well.
In genome editing the high narrow sense heritability of HPFH led to a GWAS that led to a target that led to a curative treatment strategy for sickle.
It may be messy but it proved useful.
https://t.co/pswnVoOeXO
Sep 11, 2021 · 1 like · 18,341 followersView post →
@CrisprMemes All true.
That said - Victoria Gray was dosed with ~2e8 of her own HSPCs plain-vanilla-DSBed, 80%+ edits, and 1yr later her marrow has no loss of indels.
They've dosed 30 subjects this way.
So.
We can actionably collaborate w pizza oven.
https://t.co/pswnVoOeXO
Aug 19, 2021 · 2 likes · 18,341 followersView post →
AM
ALI MUHAMMED ~
@BinMohammed07 · X
Further reading;
Aug 12, 2021 · 2 likes · 2,309 followersView post →
JK
JangKeun Kim
@KJangkeun · X
CRISP-based therapy for Sickle Cell Disease and β-Thalassemia
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia CTX00... https://t.co/Ts2y74KnfK via @YouTube
And here it is.
The possibility of transfusion independence for individuals with sickle cell anemia.
One can only imagine the possibilities of impact.
https://t.co/SwbvtbXe9o
@NaturePortfolio @Nature Wondering if someone would mind explaining how this work differs significantly from what others have done using zinc fingers and crispr-cas9 already in HSCs for sickle cell?
https://t.co/qaOvsHIzhe
https://t.co/33FGUPMnCw
https://t.co/U1Yin3sCMd
https://t.co/5uEUlaIeZX
RT @GeneticCouns: Future directions for treatment of hemoglobinopathies: This study reports on the first two patients of a CRISPR-Cas9 gene…
May 14, 2021 · 2 shares · 212 followersView post →
DC
Dr. Julián Campos
@GeneticistMx · X
RT @GeneticCouns: Future directions for treatment of hemoglobinopathies: This study reports on the first two patients of a CRISPR-Cas9 gene…
May 14, 2021 · 2 shares · 1,337 followersView post →
GC
Genetic Counselors
@GeneticCouns · X
Future directions for treatment of hemoglobinopathies: This study reports on the first two patients of a CRISPR-Cas9 gene editing clinical trial. Read more: https://t.co/ofJInwlukX #GCChat
May 14, 2021 · 4 likes · 2 shares · 14,700 followersView post →
SP
Stephan Grupp MD PhD
@GruppSteve · X
Deets on the CRISPR trials
see ASH 2020 talk by @hfrangoul and
https://t.co/Xs9gIeGfq9
May 7, 2021 · 8 likes · 1 share · 11,812 followersView post →
SZ
Stefan Zukin
@StefanZukin · X
@dsdamato "the first-ever clinical trial of the treatment in humans"
The UC CRISPR trial isn't the first. There are treatments that have already gone into humans and results have been published.
https://t.co/AXcizKZvSB
https://t.co/A3tOEyY1ne
Nutshell #218: CRISPR-Cas9 Gene Editing has enabled transfusion independence and elimination of vaso-occlusive episodes in patients suffering from Sickle Cell Disease and β – Thalassemia.
@NEJM
https://t.co/shcKe5D0SX
@ShaharTaiber @AnerOtto @RackMada זה הקפיץ ועניין אותי, כי כבר כמה שבועות אני יושב לרוקי על הראש שיעשה שרשור על המאמר של התוצאות הראשוניות מניסוי משלב 1+2 בקריספר לטיפול באנמיה חרמשית ובטא טלסמיה, והוא כל פעם מתחמק מזה
Feb 21, 2021 · 2 likes · 1,763 followersView post →
AK
Andrew Kernytsky
@kernytsky · X
@JialiangLiang @Biotech2k1 Hmm... We looked for off-target in CRISPR's lead program and didn't detect any. Guess I'm not an everybody... https://t.co/Y92q3shh4B
@Joner_MD Men altså. Der er gode use-cases derude: https://t.co/BVAgqXgjEi og https://t.co/ZVsSwgFpGs. I Danmark er det @rasmus_o_bak der er CRISPR troldmand og ham der kommer til at redde patienter med teknikken.
Feb 12, 2021 · 1 like · 2,300 followersView post →
PP
Paracelsus Pharmacology
@PMUpharmacology · X
Globin switching induced by CRISPR-Cas9- or lentivirus shRNA-mediated gene silencing of transcription factor BCL11A results in clinical improvement of patients with #hemoglobinopathies, two studies suggest:
https://t.co/mYWJ1eyWlD
https://t.co/cBYZ8pFBc3
Treatment by #CRISPR -Cas9 gene editing—a proof of principle in a patient with sickle cell anemia and a patient with thalassemia.
Read the editorial: https://t.co/T6EOa20Sjf
And the article: https://t.co/roLicyZFxQ https://t.co/4wNihQTijf
After the breakthrough of mRNA vaccines now CRISPR technology being used to cure sickle cell anemia and beta-thalassemia. These two technologies will revolutionize the face of medicine forever.
#CRISPR
#SickleCell
#betathal
https://t.co/usnai0TxVZ
Linus Pauling announced the first molecular disease 70 or so years back, and now gene therapy based on enhancer function of BCL11A. Great progresses based on so many discoveries.
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia | NEJM https://t.co/MjYjMC0joB
RT @CARTTherapy: CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia | NEJM https://t.co/r4gqhVuNLK
Jan 22, 2021 · 2 shares · 2,103 followersView post →
MM
Matthew Sparks, MD
@Nephro_Sparks · X
RT @NephRodby: @Nephro_Sparks, no longer a @NephMadness pipe dream? This is VERY EXCITING. https://t.co/1UbVtXuxBS
Jan 22, 2021 · 1 share · 21,165 followersView post →
RR
Roger Rodby
@NephRodby · X
@Nephro_Sparks, no longer a @NephMadness pipe dream? This is VERY EXCITING. https://t.co/1UbVtXuxBS
Jan 22, 2021 · 9 likes · 1 share · 16,050 followersView post →
CN
Cell Therapy News
@celltherapynews · X
#CRISPR technology to cure sickle cell disease at @thisisUIC!
Researchers reported #GeneEditing modified the DNA of stem cells by deleting the gene BCL11A. @NEJM
Jan 22, 2021 · 1 like · 12,205 followersView post →
I'm late to the party on this one but - what a milestone!
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia | NEJM https://t.co/3fvfGH5ehg
Two trials show promising results with gene therapies that target sickle-cell anemia
https://t.co/i5P0W87sFO
https://t.co/g3JUkuPqPZ
https://t.co/IMvY9bZ0su https://t.co/RZAarNPwXt
RT @ImmunoEditor: Happy New Year! In today's issue of @ScienceMagazine, I highlight a recent article by @SelimCorbaciog1 & colleagues in @N…
Jan 1, 2021 · 2 shares · 1,000 followersView post →
PM
Patrick McGuire
@McGuirePatrick · X
RT @ImmunoEditor: Happy New Year! In today's issue of @ScienceMagazine, I highlight a recent article by @SelimCorbaciog1 & colleagues in @N…
Jan 1, 2021 · 2 shares · 1,122 followersView post →
SS
Seth Thomas Scanlon
@ImmunoEditor · X
Happy New Year! In today's issue of @ScienceMagazine, I highlight a recent article by @SelimCorbaciog1 & colleagues in @NEJM describing the use of #CRISPR to treat 2 different human blood disorders!
EC: https://t.co/1NOWsCWrmo
Article: https://t.co/e2pxqUOgaq https://t.co/7zDQiuRCoq
Jan 1, 2021 · 11 likes · 2 shares · 3,346 followersView post →
EO
emeka_okafor
@emeka_okafor · X
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia https://t.co/J1GOZOuOyX
RT @emlitofnote: In some parallel universe without #covid19, these stories of gene-silencing and gene-editing in @nejm to *cure* sickle cel…
Dec 13, 2020 · 1 share · 13,681 followersView post →
RM
Ryan Radecki, MD MS
@emlitofnote · X
In some parallel universe without #covid19, these stories of gene-silencing and gene-editing in @nejm to *cure* sickle cell disease would be getting top billing.
Nearly miraculous, even in these nascent stages of development.
https://t.co/DhwSFwsnlG
https://t.co/9q3KCZQyZv https://t.co/NtuDz5anWD
Dec 13, 2020 · 7 likes · 1 share · 14,294 followersView post →
VR
Vardit Ravitsky
@VarditRavitsky · X
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia | NEJM https://t.co/uFQCBsj6hD
Dec 12, 2020 · 1 like · 3,988 followersView post →
OG
Ognjen Gajic
@ogi_gajic · X
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia | NEJM https://t.co/arRSRYlb2W
Dec 11, 2020 · 1 like · 1 share · 5,960 followersView post →
MB
Maddison Bartlett
@MaddisonBartle2 · X
RT @dalchas: Great news for SCD and TDT: CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia | NEJM https://t.co/fPvGO24bTv
#CRISPR-Cas9 #Gene Editing for Sickle Cell Disease and β-Thalassemia | NEJM https://t.co/K5gh7iobpr
Dec 10, 2020 · 4 likes · 3 shares · 4,507 followersView post →
NB
Nam Bui
@NamBuiMD · X
Innovative CRISPR-based approach (in humans!) to treat sickle cell/B-Thal by increasing HbF via downregulation of BCL11A. Although you do need a myeloablative transplant...
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia | NEJM https://t.co/yl3O7AmEma
Paper of the Month (maybe century) “CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia” in @NEJM. Gene editing had many positive outcomes including transfusion independence and elimination of vaso-occlusive episodes! #HemeTwitter https://t.co/5aDkPEpqhq
Dec 8, 2020 · 6 likes · 2 shares · 4,822 followersView post →
T
Thuo
@ThuoPap · X
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. https://t.co/VIB03uZVzy
Exagamglogene autotemcel and Glutamine Interactions - Drugs.com
News
DOI: 10.1056/NEJMoa2031054 https://www.nejm.org/doi/suppl/10.1056/NEJMoa2031054/suppl_file/n. Food/lifestyle interactions. No alcohol/food interactions were ...
The Next Growth Phase of Gene Editing The gene editing market is ...
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DOI: 10.1056/NEJMoa2031054. 2. Liu, Y., Cheng, Z., Wei, G., et al. (2024). Advances in Base Editing: A Promising Tool for Therapeutics. Nature Biotechnology ...
What next for CRISPR after first therapeutic approvals?
News
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. New England Journal of Medicine, 384(3), 252–260. https://doi.org/10.1056/nejmoa2031054. Li, ...
org/doi/full/10.1056/NEJMoa2031054 · May be an image of text that says 'မိုမီ rosehill hill LIFE SCIENCES 5 Molecules · May be an image of text that says ...
A Brief History of CRISPR-Cas9 Genome-Editing Tools
Blogs
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. N Engl J Med. 2021 Jan 21;384(3):252-260. doi: 10.1056/NEJMoa2031054. Epub 2020 Dec 5. PMID: ...
... 10.1056/NEJMoa2031054 · godlords. •. לפני שנה (1). השתלת מח עצם, בעריכה במבחנה. אספקה לכל החיים של מדכאי חיסון כדי לאפשר להם להתרבות. ככה עריכת ...
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FF
VG
JG
DB
CC
B
IM
SA
JE
יק
IS
SP
TS
DR
C
G
Y
دا
FU
KF
AM
JK
TA
UM
E
PP
WI
A
N
SJ
AP
MC
DC
GC
SP
R
SM
SZ
HB
SY
À
AK
MB
PP
DP
VP
AH
MM
RV
AE
KI
FC
PV
EM
CN
AB
MA
MM
RR
CN
CR
US
TP
DA
C
RL
O
KC
AB
AH
HL
PM
SS
EO
BB
UB
DR
L
S
GL
VP
T
SN
C
DA
RM
VR
OG
MB
CV
ME
AB
NB
RB
JK
JB
BM
RP
モ
OM
DM
IP
DR
O
NH
T
TT
PH
EC
GE
DJ
FA
JM
W
GR
ET
C
KL
JG
NM
TD
SA
YA
RB
P
CS