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Tag Directory / GENETHERAPY     showing 1–4 of 4   RSS



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Stem cells have potent potential for diabetes treatment

medicalxpress - Humans have around 30 trillion cells in our adult bodies. Amazingly, each of these cells came from a handful of about 100 stem cells in the earliest days of development. The ability of these embryonic stem cells to turn into any cell type makes them pluri…

AI Summary: Researchers report stem cell–based approaches can replenish insulin‑producing cells and restore glycemic control in diabetes models, offering a potential path beyond daily insulin injections. Early findings suggest significant therapeutic promise, but scientists stress that safety, durability, and immune‑rejection hurdles must be cleared before these techniques graduate from experimental hope to standard care.

#healthcare #pharmaceuticals #biotech #drugdevelopment #clinicaltrials #diabetes #genetherapy

5 days / medicalxpress

7 days / livescience

8 days / medicalxpress


Back to Top / Saturday, May 23, 2026, 4:21 pm / permalink 24259 / 4 stories in 8 days /

Data-Driven Decision Support in Obesity Management Commission: enabling more equitable and personalized obesity care

Paul W. Franks / nature - Nature Medicine, Published online: 12 May 2026; doi:10.1038/s41591-026-04363-0Announced in this Comment and in collaboration with Nature Medicine is the convening of the Data-Driven Decision Support in Obesity Management Commission, to promote adequate sc…

AI Summary: A phase 1/2 study of CRISPR‑Cas9 CD33‑deleted allogeneic hematopoietic cell transplantation followed by gemtuzumab ozogamicin maintenance reports encouraging early signals in AML. The gene‑editing approach aims to protect donor cells from CD33‑targeted therapy, potentially enabling safer post‑transplant maintenance and offering a novel strategy to marry cellular engineering with targeted antibody therapy.


CD33‑targeted transplant and post‑transplant maintenance

8 days / oncodaily

11 days / oncodaily

14 days / oncodaily

19 days / medicalxpress


Safety and ethical scrutiny of gene editing and gene therapy

9 days / oncodaily


Scaling cell therapy: accreditation and expanding CAR indications


All Other Stories

2 days / esmo

3 days / nature

3 days / newscientist

6 days / oncodaily

6 days / oncodaily

6 days / medicalxpress

7 days / oncodaily

7 days / oncodaily

8 days / oncodaily



Preclinical evaluation of antisense oligonucleotide therapy in a mouse model of HNRNPH2-related neurodevelopmental disorder

Ane Korff, Xiaojing Yang, Ozan Ozdemir, Ananya Samanta, Yong-Dong Wang, Tushar Patni, Alfonso J. Lav / science - Science Translational Medicine, Volume 18, Issue 846, April 2026.

AI Summary: Researchers report that antisense oligonucleotide therapy reversed neurological deficits in mouse models of HNRNPH2‑related neurodevelopmental disorder. The preclinical results provide a targeted mechanism to correct pathogenic RNA processing, moving a once‑untreatable condition toward a plausible therapeutic path — pending the usual caution about translating mice to humans.

#pharmaceuticals #biotech #drugdevelopment #infanthealth #behavioralhealth #genetherapy #genetics

23 days / medicalxpress


Back to Top / Monday, April 27, 2026, 3:21 am / permalink 22803 / 5 stories in 4 wks /

Gene therapy improves hearing in 90% of patients with inherited deafness in largest trial of its kind

livescience - A new gene therapy tested in China has improved the hearing of 38 people who were born deaf due to mutations in a gene called OTOF.

AI Summary: A gene therapy for inherited deafness delivered dramatic results, restoring hearing in roughly 90% of treated patients in the largest trial of its kind. Investigators report durable improvements over follow-up, signaling a potential one-time intervention for certain genetic deafness types and challenging the notion that auditory loss is always irreversible. Hope, meet hard data.


FDA approves first-ever gene therapy for inherited hearing loss

5 wks / livescience


Primate study finds human-like genetic cause of blindness


Trial shows durable hearing restored in most patients

5 wks / livescience



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