CRISPR workshop starts TOMORROW.
sgRNA design → off-target analysis → base editing → prime editing → HDR templates → Cas protein structure → clinical applications.
ACT NOW 👇
bdglifesciences.com/event/cr…
#CRISPRCas9 #GeneTherapy #GenomeEditing
This study demonstrates that tRNATrp.UAG - a novel engineered tRNA treatment can mitigate vision loss in models of Leber congenital amaurosis, highlighting a promising therapeutic approach for inherited retinal degenerative diseases. @UWMadison
#STTT #OpenAccess: doi.org/10.1038/s41392-026-0…
#GeneTherapy #RetinalDisease #MolecularMedicine #RareDisease #Genomics #BiomedicalResearch
ALT Engineered tRNA reduces vision loss in a mouse model of Leber congenital amaurosis
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A century from now, historians of medicine may look back at this moment the way we look at the first heart transplant or the discovery of antibiotics.
For the first time, a human has received a therapy designed not just to treat disease, but to restore a more youthful cellular state.
The science is called partial epigenetic reprogramming.
The ambition is extraordinary: to help aged cells remember how to function like younger versions of themselves.
No, this is not age reversal.
No, we don’t know if it will work.
But for the first time, the question is no longer theoretical.
A human trial has begun.
The age of testing rejuvenation medicine in people has officially started.
#Longevity #Biotechnology #GeneTherapy #FutureOfMedicine
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🧬 من الرعاية الداعمة إلى تعديل الجينات: كيف غيّر العلاج الجيني مستقبل طب الأعصاب
بقلم: أ.د. أحمد باماقا @abamaga
العلاج الجيني لم يعد فكرة مستقبلية… بل أصبح واقعًا يغير حياة المرضى. مقال يستعرض التحول الكبير نحو استهداف مسببات المرض من جذورها.
🔗 ssmg.org.sa/blog/3251
#GeneTherapy #GeneticMedicine #FutureOfMedicine
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Exploring advanced therapeutics abroad? Accessing gene therapy pathways in China requires precise record translation and strict institutional screening.
Read our full guide: zurl.co/C3XkJ
#MedicalTourismChina #GeneTherapy #MedicalConcierge #MedBridgeNZ
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That's a significant advancement in #GeneTherapy! Delivering full-length mRNA without viral vectors could reduce immune reactions and potentially enhance treatment efficacy. I'm curious how this platform compares in terms of efficiency and safety to existing methods. Dr. Betty Kim's work sounds promising for the future of #Medicine. For anyone interested in exploring more about breakthroughs in the biomedical field, Sci-Quest is a one-stop platform for all your questions, offering capabilities to generate comprehensive biomedical reviews. Check it out: [sciqst.com](sciqst.com) #Biotech #Innovation
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RealityB retweeted
Jun 11
#geneTherapy 1st generation treatments from $NTLA $CRSP $BEAM $PRME $crbu $rgnx $SRPT $QURE are poised to double as #POTUS pushes #IranWar confrontation to the final mile
$XBI $arkG #healthcare #InPlay post #IranWar
Jun 10
$PRME CEO explains why the CGD program was revived:
1⃣FDA meaningfully relaxed CMC requirements last year to better support ultra-rare diseases
2⃣Agency agrees that the two treated patients’ outcomes are strong enough to support approval
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Jun 13
🚨 Game-changer for Retinitis Pigmentosa patients!
Ocugen’s OCU400 — the first gene-agnostic modifier gene therapy for RP — just received major EMA support:
✅ Positive ATMP classification
✅ Phase 3 trial design accepted for European Marketing Authorization Application !
Phase 3 enrollment complete. MAA filing planned 1-3 Months after BLA filling → potential conditional approval in Europe H2 2027?
A one-time treatment that could help patients across dozens of genetic mutations. Real hope for a disease with almost no options. 👁️🧬
Repost it, if this gives you hope for gene therapy!
#OCU400 #RetinitisPigmentosa #GeneTherapy #Ocugen #EMA #Blindness #Biotech #OCU400 #RetinitisPigmentosa #GeneTherapy #Ocugen #EMA
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حنان محرق 🌹 retweeted
معلومات وراثية 🧬 في أقل من دقيقتين و كذلك حول آلية تصحيح الطفرات عن طريق #العلاج_الجيني 🧬
#Genomics #Gene #GeneTherapy
#الجينوم_البشري
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Jun 13
🧬 Can CRISPR cure hemoglobinopathies in children?
New data from the CLIMB THAL-141 and CLIMB SCD-151 trials, published concomitantly with #EHA26, are remarkable.
🩸 β-thalassemia
✅ 8/8 evaluable children achieved transfusion independence
🩸 Sickle cell disease
✅ 8/8 evaluable children remained free of severe vaso-occlusive crises
Fetal hemoglobin rose rapidly and remained durable, with normalization of hemoglobin levels.
⚠️ Not without risk:
All patients experienced Grade 3-4 adverse events, and 1 treatment-related death occurred due to busulfan-associated veno-occlusive liver disease.
These early results suggest CRISPR-based gene editing may offer a potentially curative option before irreversible organ damage develops.
Will gene editing become the preferred curative strategy over allogeneic transplant in pediatric hemoglobinopathies?
@NEJM @VertexPharma @CRISPRTX @oncoalert @myESMO @ASCO
#EHA26 #CRISPR #GeneTherapy #SickleCellDisease
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🧬🎯 Harnessing engineered ribozymes for precision cancer therapeutics!
📖Read more at: mdpi.com/2674-0583/3/4/20
#SyntheticBiology #CancerResearch #GeneTherapy #RNABiology #Ribozymes #PrecisionMedicine #Biotechnology #Leukemia #MolecularMedicine
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💬 “I think ICLE is the top conference for scientists and physicians who are interested in the engineering of lymphocytes, for the therapy of cancer, autoimmune disease, and beyond.” - @barzel_adi
Register now: lymphocyte.kenes.com/registe…
#Immunotherapy #GeneTherapy #CellTherapy
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Gene Therapy for Wound Healing: The LEGenD-1 Trial #ActAgainstAmputation #Innovation #GeneTherapy diabeticfootonline.com/2025/…
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Unlock advanced AAV capsid characterization by joining our expert-led GEN webinar series with @uspharmacopeia. Register free: hubs.li/Q04jFgB-0 #AAV #GeneTherapy #Biotech
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Jun 12
Researchers at @UCSanDiego are working on #genetherapy that prevents the TDP-43 protein from developing, which influences the development of neurodegenerative diseases. More research is needed, but this therapy shows promise. today.ucsd.edu/story/experim…
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Excited to share that Pauline Skigin and Simon Dumolard presented their work at the PGBS Symposium 2026 in Basel on June 11, focused on advances in gene therapy.
Many thanks to Dr Jens Casper for the invitation!
#GeneTherapy #LNPs #siRNA #drugdelivery
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