Northwestern Medicine scientists have identified the cause of a genetic subtype of autism and schizophrenia that results in social deficits and seizures in mice and humans. Scientists have discovered ...
The FDA has awarded U.S. orphan drug designation and rare pediatric disease designation to Huidagene Therapeutics Co. Ltd.’s HG-204 (AAV-hfCas13Y-gMECP2), an RNA editing therapy based on CRISPR/Cas13Y ...
Researchers have uncovered how specific DNA rearrangements called inverted triplications contribute to the development of various genetic diseases. The study reveals that during DNA repair, segments ...
The FDA has awarded U.S. orphan drug designation and rare pediatric disease designation to Huidagene Therapeutics Co. Ltd.’s HG-204 (AAV-hfCas13Y-gMECP2), an RNA editing therapy based on CRISPR/Cas13Y ...
Brantley was born with MECP-2 Duplication Syndrome. Many Americans first learned about this progressive, neurological disorder in 2018, when NBC’s Richard Engel first announced this his son had been ...
Findings bolster the idea that the functions of this protein -- MeCP2 -- are more centered on nucleosomes, rather than other forms of DNA. One particular protein lies at the heart of brain development ...
SHANGHAI and CLINTON, N.J., Oct. 31, 2023 /PRNewswire/ -- HuidaGene Therapeutics (辉大基因，"HuidaGene"), a global clinical-stage biotechnology company focused on developing CRISPR-based programmable ...
A newborn male with Fryns syndrome (FS), a lethal autosomal recessive condition, also had mosaic tandem duplication of chromosome 1q. Multiple malformations were noted at birth including diaphragmatic ...
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