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 ...
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 ...
Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic ...
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 ...
A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If ...
Experimental gene therapies like AMT‑130 lower toxic huntingtin protein and significantly slow Huntington’s disease, offering families new hope for longer, better quality lives.
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 ...
Regeneron has posted updated clinical data on DB-OTO, providing more evidence of the gene therapy’s potential to treat a rare genetic form of deafness as it plans for a potential FDA filing later this ...
Gene therapy achieved its first major breakthroughs in the early 1990s with the treatment of a rare disease called severe combined immunodeficiency. However, the field was dealt a serious setback in ...
Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts treated with cochlear implantation in a cohort study from China. Gene ...
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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