New gene therapy cures 1st New Yorker of sickle cell anemia
The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely ...
Gene therapy, the idea of fixing faulty genes ... Scientists Discover New Code Governing Gene Activity Aug. 20, 2024 — A newly discovered code within DNA -- coined 'spatial grammar' -- holds a ...
In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...
Danon disease is a rare but serious cardiomyopathy that drastically reduces life expectancy. Male patients infrequently live ...
New findings show that the mitochondria powering our cells also control the ability of a DNA repair protein to suppress the senescence-associated secretory phenotype (SASP), which causes zombie-like ...
The one-time therapy appears to correct the mutation that causes the disease, known as Alpha-1 antitrypsin deficiency (AATD), ...
Typically, as a person gets older, the gapping of the end of the telomere chromosomes gets shorter, allowing DNA strands to separate ... Elsewhere in the field of gene therapy for rare disorders ...
While initial study results suggest Beam's technology can correct alpha-1 antitrypsin deficiency's genetic roots, shares fell ...
With MeiraGTx eyeing up how to take the drug through phase 3 and onto commercialization, Hologen has stepped in. The ...
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