Australian cancer researchers are the first to establish a next-generation gene-editing tool for modeling and interrogating human disease.

Findings indicate that altering glucose metabolism in T cells boosts their therapeutic potential against melanoma, paving the ...

Experts hailed exciting news for some people with sickle cell disease in England as the health watchdog decided to grant ...

Charlestown, Massachusetts Thursday, January 23, 2025, 15:00 Hrs [IST] ...

The malfunctioning mitochondria seem to poison the immune cells, explains Yosuke Togashi, a pulmonary physician and ...

Only dual route gene transfer therapy in development to treat Friedreich’s ataxia with FDA IND clearance and Fast Track designation ...

clinical trials for various mitochondria-targeted therapies should be conducted to confirm the therapeutic efficacy of natural products discussed in this review. The trials should integrate a wide ...

the company’s investigational gene therapy for Leber Hereditary Optic Neuropathy (LHON) due to a mutated ND4 mitochondrial gene. The patients had all participated in the phase III trials RESCUE and ...

Gene therapy has been available since 2022 for hemophilia B and since 2023 for hemophilia A, yet some major medical institutions have barely treated any patients so far. What’s the holdup?

is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based ...

It may be, she added, that weekly therapy sessions are simply not scalable to a broad population, and the field should explore light-touch alternatives, like single-session interventions and ...