FDA vaccine chief Vinay Prasad leaves following criticism from Laura Loomer
WASHINGTON (AP) — The Food and Drug Administration’s polarizing vaccine chief is leaving the agency after a brief…
Rocket’s PKP2-Arrhythmogenic Cardiomyopathy Gene Therapy RP-A601 Snags RMAT Designation
Rocket Pharmaceuticals’ RP-A601, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat plakophilin-2 related Arrhythmogenic Cardiomyopathy…
Breakthrough gene therapy reverses genetic deafness across age groups
Using gene therapy to treat hereditary deafness is safe and effective in both children and adults, according to…
Molecular genetics of J-domain protein-related chaperonopathies in skeletal muscle
Kampinga HH, Andreasson C, Barducci A, Cheetham ME, Cyr D, Emanuelsson C, et al. Function, evolution, and structure…
Sequencing validates deep learning models for EHR-based detection of Noonan syndrome in pediatric patients
Cohort characteristics and model risk score distribution The study cohort comprised 92,493 patients enrolled in the DT Biobank…
Nanoscope Begins FDA Submission for Breakthrough Retinitis Pigmentosa Therapy
Dallas-based Nanoscope Therapeutics has taken a major step toward offering new hope to people who are losing their…
These Genetically Modified Mice Make Their Own ‘Ozempic’
The popularity of Ozempic and Wegovy as diabetes and weight loss medications has skyrocketed. But how far would…
Genetic and oncogenic features of RASGRF fusions
Prevalence of RASGRF fusions in solid tumor malignancies We previously reported three RASGRF1 fusions: (1) an OCLN–RASGRF1 fusion…
Molecular genetics of myotonic dystrophy and the evolution of therapeutic approaches
Nakamori, M & Takahashi, MP Myotonic dystrophy. In: Takeda S, Miyagoe-Suzuki Y, Mori-Yoshimura M, editors. Translational research in…
AAV gene therapy for autosomal recessive deafness 9: a single-arm trial
Chang, Q. et al. Virally mediated Kcnq1 gene replacement therapy in the immature scala media restores hearing in…