The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...
Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic ...
AVALA Hospital, in partnership with Retina Associates New Orleans, has successfully performed Louisiana’s first ENCELTO procedure, a cell-based gene therapy for adults with macular telangiectasia type ...
A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If ...
A revolutionary treatment brings new hope to those suffering from a devastating rare skin disorder, while showcasing an innovative funding model that could transform rare disease research In a ...
Experimental gene therapies like AMT‑130 lower toxic huntingtin protein and significantly slow Huntington’s disease, offering ...
NHS Scotland has approved a one-time CRISPR gene therapy for severe sickle cell disease, offering durable reduction in vaso-occlusive crises for eligible patients.
US biotech firm Fractyl Health is developing Rejuva, an experimental gene therapy that may mimic GLP-1 drugs for years after ...
There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University's College of Medicine and the UMass Chan Medical School have achieved ...
UniQure Gene Therapy’s Clinical Trial Results Keep It on Path to Become First Huntington’s Treatment
A uniQure gene therapy slowed progression of Huntington’s disease by 75% after three years, statistically significant clinical trial results the company says will support plans for a regulatory ...
Fractyl Health is developing a shot that would program the body to make more of the GLP-1 hormone naturally, a risky bet that it can provide a longer-lasting benefit than blockbuster weight-loss drugs ...
A Spanish study shows that patients with genetic dilated cardiomyopathy who experience severe arrhythmias are at an elevated risk of developing advanced heart failure and requiring a heart transplant.
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