GEN - Genetic Engineering and Biotechnology News17h
From Tools to Trials: Editing Therapy Companies Pivot to Development
A pair of privately-held developers of editing-based therapies have some successes to show for their recent efforts to ...
Breakthrough gene therapy alleviates Dravet syndrome symptoms in mice without side effects
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
BioWorld2d
CMV history cited in DMD liver death on Sarepta’s Elevidys
Wall Street was weighing the gravity of the death from acute liver failure of a patient who was treated for Duchenne muscular dystrophy (DMD) with Sarepta Therapeutics Inc.’s gene therapy, Elevidys ...
GEN - Genetic Engineering and Biotechnology News1d
Gene Therapy Tested in Mice Offers New Hope for People with Dravet Syndrome
By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without ...
EurekAlert!2d
Breakthrough Dravet syndrome gene therapy in mice brings new hope to families
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
Science Daily6d
Secret boss of the liver: Star-shaped cells that promote fibrosis also regulate liver health
New research suggests restoring stellate cells to their healthy and protective state could simultaneously reduce liver scarring and improve liver function.
MedCity News2d
Liver Failure-Associated Death Reported in Patient Treated With Sarepta Gene Therapy Elevidys
Liver injury is a known complication risk of Elevidys and other gene therapies that use an adeno-associated (AAV) virus to deliver the one-time treatment to cells. That risk is already flagged on ...
pharmaphorum1d
Longevity: Anti-ageing drugs, watch out, here we come…
The company has made significant progress and plans to file an IND for its lead compound, GF-1002, an exo-AAV expressing cDNA of centenarian variant sirtuin 6 (SIRT6), for the treatment of ...
ahajournals.org3d
Nrf3-Mediated Mitochondrial Superoxide Promotes Cardiomyocyte Apoptosis and Impairs Cardiac Functions by Suppressing Pitx2
Global (Nrf3-KO) and CM-specific (Nrf3 CM) Nrf3 knockout mice were subjected to MI or ischemia/reperfusion ... MitoParaquat administration and CM-specific adeno-associated virus vectors were used to ...