gene therapy
Dilated Cardiomyopathy Gene Mutations Point to Possible Therapeutic Strategy
Homozygous loss-of-function mutations in the BAG5 gene turned up in a subset of inherited DCM patients, and BAG5 appeared amenable to gene therapy in mouse models.
Investigational CRISPR Therapy Shows Efficacy Against Transthyretin Amyloidosis in Phase I Study
The therapy works by reducing serum concentration of misfolded transthyretin protein, which leads to the life-threatening disease.
Mission Bio Seeks to Increase Drug Trial Collaborations, Improve Gene and Cell Therapy Efficacy
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Mission Bio will use a recent $70 million funding round to develop quality control assays using its Tapestri single-cell analysis platform.
Personalis, Sarepta Therapeutics Exploring Immune Response to Precision Genetic Therapeutics
The companies are applying Personalis' technology to explore immune response to genetic therapeutics in Sarepta's pipeline.
The Decade in Genomics: CRISPR, Single Cells, Long Reads, Clinical and Consumer Applications
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GenomeWeb asked a number of prominent researchers in the field via email about their top picks for notable developments or achievements in genomics during the 2010s.
Jul 24, 2019
Jan 11, 2017
Oct 20, 2016
Apr 28, 2009
Apr 24, 2009
Apr 24, 2009
Egyptian Biotech Group Seeks 'Omics Partners
Mar 26, 2009
Mar 5, 2009
Papers of Note for Feb 2009
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Feb 24, 2009
Feb 19, 2009
Feb 5, 2009