Prime editing corrects a childhood genetic disease in mice
Alternating hemiplegia of childhood (AHC) is a rare, one-in-a-million genetic disease that affects kids. Most cases are…
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…
Rescued by fat bubbles: Scientists treat rare genetic disease with designer molecule | Lifestyle
Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with…
Targeted in vivo gene integration of a secretion-enabled GLP-1 receptor agonist reverses diet-induced non-genetic obesity and pre-diabetes
Plasmid construction The Exe4 gene was codon-optimized using the GeneSmart codon optimization tool (https://www.genscript.com/gensmart-free-gene-codon-optimization.html) based on the amino…
Gene therapy brings back hearing in young and adult deaf patients
Gene therapy can improve hearing in children and adults with congenital deafness or severe hearing impairment, a new…
Review of 40 genes causing congenital myasthenic syndromes
Ohno K, Ohkawara B, Shen XM, Selcen D, Engel AG. Clinical and pathologic features of congenital myasthenic syndromes…
Integration of MRI radiomics and germline genetics to predict the IDH mutation status of gliomas
An overview of the study design and analysis is provided in Fig. 1. Demographic and clinical characteristics of…
SpliceBio Secures $135 Million Series B Financing to Advance Lead Program SB-007 in Stargardt Disease and Expand Pipeline of Genetic Medicines
Financing co-led by new investors EQT Life Sciences and Sanofi Ventures, with participation from Roche Venture Fund, as…
The impact of public insurance on RRSO for HBOC in Japan: a nationwide data study
JOHBOC Registration Committee: Masami Arai (Department of Clinical Genetics, Juntendo University, Graduate School of Medicine, Tokyo, Japan), Seigo…
Scientists call for 10-year ban on CRISPR for germline gene editing
Megan Molteni reports on discoveries from the frontiers of genomic medicine, neuroscience, and reproductive tech. She joined STAT…