Penn and CHOP will test gene therapy for rare diseases with a new FDA trial protocol

Earlier this year, researchers at CHOP and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a rare liver disorder. KJ Muldoon ...

Can a One-Time Gene Therapy Lower Cholesterol?

Researchers report early but encouraging results on an experimental gene-editing treatment for high cholesterol.

First-in-human trial of CRISPR gene-editing therapy safely lowers cholesterol and triglycerides

In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol ...
TCTMD

CRISPR-Cas9 Gene-Editing Therapy Shows Early Promise for Dyslipidemia

The approach is “very exciting,” but one expert says a lot more research is needed, especially around safety, before clinical ...
The Sentinel

Can gene therapy be harnessed to fight the AIDS virus?

For more than a decade, the strongest AIDS drugs could not fully control Matt Chappell's HIV infection. Now his body controls it by itself, and researchers are trying to perfect the gene editing that ...

FDA adds strongest warning to Sarepta gene therapy linked to 2 patient deaths

The Food and Drug Administration is adding a new warning to a gene therapy linked to two patient deaths earlier this year. Sarepta Therapeutics' Elevidys will carry the FDA's strongest warning ...
New Atlas

One-off lipid-lowering gene therapy a success in world-first human trial

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn ...
Fierce Biotech

Lilly bags eye-catching retinal disease gene therapy from MeiraGTx in $475M deal

MeiraGTx is cashing in on a gene therapy that restored vision to 11 children who were born legally blind, licensing the ...

Sarepta Duchenne gene therapy gets strongest FDA label after patient deaths

The FDA has restricted the number of patients who can receive the Cambridge drugmaker's gene therapy for Duchenne muscular ...
The Conversation

Gene therapy can be less effective in women – and my research in mice brings us one step closer to understanding why

Alison Clare has received funding from the Wellcome Trust and the Medical Research Council. Her position is currently funded by National Institute for Health and Care Research (NIHR) Biomedical ...

Researchers to test gene therapy for rare diseases with a new FDA trial protocol

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a ...