Explore how researchers are using next-generation CRISPR therapies to target the root cause of muscle degeneration.
TF (transcription factor) Prdm16 (positive regulatory domain–containing protein 16) regulates hematopoietic and neuronal stem cell homeostasis, adipose differentiation, and cardiac development. Its ...
A team led by Cedars-Sinai Health Sciences University investigators has created a faster, cheaper way to determine the genes expressed in cancerous tumors. The AI-based tool, which they describe in ...
Circular RNAs (circRNAs), a conserved class of non−coding RNAs, are hypothesized to play functional roles in diabetic retinopathy (DR), yet their expression landscape and molecular involvement in ...
Physician and molecular biologist Katherine High remembers sitting at a staff meeting of the gene therapy company Spark Therapeutics on November 15, 2018, waiting to hear from a guest speaker, when ...
The publication of the draft human genome in 2001 introduced a post-genomic era that promised global resolution of the molecular landscape in both preclinical and clinical research environments 1. To ...
The US Food and Drug Administration on Thursday approved the first gene therapy for inherited hearing loss, a one-time treatment that proved to be life-changing for a small number of children in a ...
The treatment, the first of its kind, was approved by the Food and Drug Administration on Thursday. “Our baby was born deaf, and now he can hear,” said one parent. By Gina Kolata The Food and Drug ...
An experimental gene therapy appears safe and highly effective for restoring hearing to people born with a rare form of deafness, researchers reported Wednesday. The study, the largest and longest to ...
The authors describe the scientific foundations of a study of gene therapy for hearing loss caused by damaging variants in the gene OTOF. Department of Otorhinolaryngology and Head and Neck Surgery, ...
In 2018, a nervous-looking He Jiankui took the stage at a scientific conference in Hong Kong. A hush settled over the packed auditorium as the soft-spoken Chinese scientist adjusted his microphone and ...
A new CRISPR breakthrough shows scientists can turn genes back on without cutting DNA, by removing chemical tags that act like molecular anchors. The work confirms these tags actively silence genes, ...
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