من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن من الخلايا الدم الحمراء و مستويات الهيموجلوبين
CRISPR-Cas9 Editing of the and Promoters to Treat Sickle Cell Disease.
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Aug 2023 – Aug 2024Where it's discussed
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X / Twitter99 posts
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AI synopsis
What people are saying
Several discussions emphasize the positive outcomes of the study, such as the mention of three patients being cured without off-target effects revealing a significant advancement in treatment. Terms like 'hope' and 'promising early results' are frequently used, indicating an optimistic tone that resonates throughout the discussions and highlights the potential breakthroughs in this field.
The majority of discussions express strong support for the CRISPR-Cas9 methodology and its potential applications, indicating consensus on the research's validity.
Participants demonstrate a high level of curiosity and enthusiasm for the topic, as evidenced by the numerous discussions highlighting the implications and achievements of the study.
While many posts celebrate the findings, only a few deeply analyze the science, suggesting moderate engagement overall.
There is a prevalent belief that the publication could have profound implications for treating sickle cell disease, as many comments reflect optimism about the future of gene therapy.
Abstract in brief
- Sickle cell disease results from a defect in the adult hemoglobin globin subunit, leading to deformed red blood cells and serious health complications.
- OTQ923 is a CRISPR-Cas9 edited therapy that disrupts globin gene promoters to increase fetal hemoglobin levels, which helps protect against these complications.
- In clinical trials, participants receiving OTQ923 showed stable increases in fetal hemoglobin levels, indicating successful engraftment and potential for long-term treatment effectiveness.
Top posts
Most liked on X and BlueskyOriginal Article: CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease https://t.co/wa3S5ZdpsA #hematology https://t.co/TUcVZhx84j
Treating #Sickle_Cell_Disease with #CRISPR CRISPR-Cas9 disruption of the HBG1 and HBG2 gene promoters was an effective strategy for induction of fetal hemoglobin in three patients. https://t.co/ktzkmY3x6V
This week: #CRISPR editing for #SickleCellDisease!
CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease: https://t.co/m688uJqBsg
Featured today by @MSKLibrary: #CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat #SickleCell Disease @AkshaySharmaMD @JJ_boelens @MariaCancio3 @janbra1 @Seredev1 @jl_labelle @NEJM https://t.co/z8xy0aIOIT
All mentions
CRISPR Advances in Treating Sickle Cell Disease with OTQ923
A new gene-editing treatment utilizing OTQ923 has shown promise in treating sickle cell disease by increasing fetal hemoglobin levels. This advancement results in reduced health complications for patients, showcasing…
Advancements in CRISPR for Sickle Cell Disease Treatment
On this episode of Healthcare Unfiltered, Dr. LaBelle discusses CRISPR-Cas9 technology's potential to treat sickle cell disease. He shares insights from his NEJM research, highlighting the promising outcomes with OTQ923…
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
Dr. LaBelle received this honor for his work on stem cell gene therapy for patients with sickle cell disease https://t.co/zqD66hlGIw
RT @CRForumUS: 2024 Top 10 Clinical Research Achievement Awards: Congratulations to James LaBelle, MD, PhD, @UChiPritzker, on the study "CR…
2024 Top 10 Clinical Research Achievement Awards: Congratulations to James LaBelle, MD, PhD, @UChiPritzker, on the study "CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease"! https://t.co/3B6OybHyGF #Top10ClinicalResearchAwards https://t.co/shNSLZalnD
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
【抄読会】 https://t.co/SlkahY1ovU 鎌状赤血球症は、ヘモグロビンを構成するβ鎖にホモ接合体で遺伝子異常があると発症する。低酸素になると赤血球が鎌状に変形するため、抹消血管内皮に付着して、血栓形成に至る。その結果として、骨、脾臓、肺、腎臓などで血管閉塞クリーゼが生じる。
Gene therapy in humans seems efficient in phase 1/2 trial for hematological disorder (technically relative simple). This calls for good EU / USA international legislation. CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease. https://t.co/pnSjJvkrwx
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @MSKLibrary: Featured today by @MSKLibrary: #CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat #SickleCell Disease @AkshayShar…
RT @MSKLibrary: Featured today by @MSKLibrary: #CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat #SickleCell Disease @AkshayShar…
RT @MSKLibrary: Featured today by @MSKLibrary: #CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat #SickleCell Disease @AkshayShar…
Featured today by @MSKLibrary: #CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat #SickleCell Disease @AkshaySharmaMD @JJ_boelens @MariaCancio3 @janbra1 @Seredev1 @jl_labelle @NEJM https://t.co/z8xy0aIOIT
https://t.co/TaswlSxbRL
What’s an interesting finding!! Three sickled patients were cured by CRISPER-Cas9 technique. No off target detected. 18 m follow up, HbF ⬆️ and sickle cell ⬇️. Congrats CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease | NEJM https://t.co/4z3MGHzebL
RT @DRKAMALAYOUB: نجاح تقنية تعديل الجينات في علاج ثلاثة مرضى ممن لديهم داء فقر الدم المنجلي الشديد وحصول استجابة سريرية لهذا العلاج ضمن در…
RT @stemcellpodcast: This week: #CRISPR editing for #SickleCellDisease!
RT @stemcellpodcast: This week: #CRISPR editing for #SickleCellDisease!
This week: #CRISPR editing for #SickleCellDisease!
RT @DrAbdulmajeedFR: من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن…
RT @DrAbdulmajeedFR: من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن…
RT @AusStemCell: 'Gene-tweaked stem cells offer hope against sickle cell disease.' Paper: https://t.co/7qQYJnmhAx https://t.co/tRVMZGcY3q
RT @NEJM: Original Article: CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease https://t.co/wa3S5ZdpsA #hemat…
RT @DrAbdulmajeedFR: من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن…
RT @DrAbdulmajeedFR: من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن…
RT @DrAbdulmajeedFR: من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن…
RT @DrAbdulmajeedFR: من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن…
RT @DrAbdulmajeedFR: من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن…
RT @DrAbdulmajeedFR: من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن…
RT @DrAbdulmajeedFR: من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن…
RT @DrAbdulmajeedFR: من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن…
RT @DrAbdulmajeedFR: من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن…
RT @DrAbdulmajeedFR: من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن…
RT @DrAbdulmajeedFR: من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن…
RT @DrAbdulmajeedFR: من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن…
RT @DrAbdulmajeedFR: من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن…
RT @DrAbdulmajeedFR: من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن…
RT @DrAbdulmajeedFR: من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن…
من الاخبار البحثية المشجعة #تقنية_كريسبر تقدم طريقة علاجية جديدة للانيميا المنجلية و ذلك باستهداف HBG1 و HBG2 مما حسن من الخلايا الدم الحمراء و مستويات الهيموجلوبين
RT @NEJM: Original Article: CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease https://t.co/wa3S5ZdpsA #hemat…
RT @NEJM: Original Article: CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease https://t.co/wa3S5ZdpsA #hemat…
RT @NEJM: Original Article: CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease https://t.co/wa3S5ZdpsA #hemat…
RT @NEJM: Original Article: CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease https://t.co/wa3S5ZdpsA #hemat…
“#CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease" Sharma A [..] LaBelle JL. N Engl J Med. 2023-08-31. https://t.co/isWWQ1eO9R NCT04443907 https://t.co/iD1H4WyDWt (Novartis Pharmaceuticals OTQ923)
RT @zacharie_lt: @NEJM article on #CRISPR treatment for #Sicklecell #SickleCellAwarenessMonth #BlackTwitter https://t.co/giKKtmMPQp
@NEJM article on #CRISPR treatment for #Sicklecell #SickleCellAwarenessMonth #BlackTwitter https://t.co/giKKtmMPQp
RT @RamonGarciaSanz: CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease | NEJM https://t.co/GJeDxciEad
RT @NEJM: Original Article: CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease https://t.co/wa3S5ZdpsA #hemat…
CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease | NEJM https://t.co/GJeDxciEad
RT @NEJM: Original Article: CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease https://t.co/wa3S5ZdpsA #hemat…
RT @NEJM: Original Article: CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease https://t.co/wa3S5ZdpsA #hemat…
RT @NEJM: Original Article: CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease https://t.co/wa3S5ZdpsA #hemat…
نجاح تقنية تعديل الجينات في علاج ثلاثة مرضى ممن لديهم داء فقر الدم المنجلي الشديد وحصول استجابة سريرية لهذا العلاج ضمن دراسة اولية . https://t.co/YywMBzUXUZ
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @NEJM: Original Article: CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease https://t.co/wa3S5ZdpsA #hemat…
CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease | @NEJM https://t.co/X0VkFUgKxW
RT @NEJM: Original Article: CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease https://t.co/wa3S5ZdpsA #hemat…
Original Article: CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease https://t.co/wa3S5ZdpsA #hematology https://t.co/TUcVZhx84j
RT @NEJM: With CRISPR-Cas9, a repressor of fetal hemoglobin expression was disrupted in hematopoietic stem cells from three persons with si…
RT @AusStemCell: 'Gene-tweaked stem cells offer hope against sickle cell disease.' Paper: https://t.co/7qQYJnmhAx https://t.co/tRVMZGcY3q
'Gene-tweaked stem cells offer hope against sickle cell disease.' Paper: https://t.co/7qQYJnmhAx https://t.co/tRVMZGcY3q
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
https://t.co/fWZxAbk0Cd
Researchers have successfully used CRISPR-CAS-9 to perform gene editing of the fetal hemoglobin gene, disrupting expression and leading to restoring of hematopoiesis. https://t.co/es7RUxeKeP
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @FilippoMontemu1: CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease | NEJM https://t.co/j44TdK3SCj This is…
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease | NEJM https://t.co/AQQJJZ15Sd
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @IdoWolf1: המהפכה הגנטית כבר כאן! בעזרת מערכת קריספר-קאס עורכים DNA של חולים עם סוג מיוחד של אנמיה תורשתית וגורמים להם לייצר המוגלובין ח…
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
https://t.co/IX3Lwec7NZ
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @ShorterLab: CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease: https://t.co/m688uJqBsg
CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease: https://t.co/m688uJqBsg
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease | NEJM https://t.co/DQ5fWXSlxw
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @IdoWolf1: המהפכה הגנטית כבר כאן! בעזרת מערכת קריספר-קאס עורכים DNA של חולים עם סוג מיוחד של אנמיה תורשתית וגורמים להם לייצר המוגלובין ח…
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
Treating #Sickle_Cell_Disease with #CRISPR CRISPR-Cas9 disruption of the HBG1 and HBG2 gene promoters was an effective strategy for induction of fetal hemoglobin in three patients. https://t.co/ktzkmY3x6V
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @IdoWolf1: המהפכה הגנטית כבר כאן! בעזרת מערכת קריספר-קאס עורכים DNA של חולים עם סוג מיוחד של אנמיה תורשתית וגורמים להם לייצר המוגלובין ח…
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @AkshaySharmaMD: All 3 patients experienced reductions in complications related to #SickleCell disease and had an increase in hemoglobin…
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
RT @AkshaySharmaMD: #PublicationAlert Incredibly proud to share our manuscript in @NEJM describing the first 3 #SickleCell dz patients who…
CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease https://t.co/OfzR6bZDpm https://t.co/194ErN3ipA
Promising early results from trials of CRISPR edited HSPCs, which were infused into participants. https://t.co/Txj7ieYCsW