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CRISPR Corrects Duchenne-Causing Mutations
Anna Azvolinsky | Apr 12, 2017
Using CRISPR-Cpf1 gene editing, researchers have fixed mutations that cause a form of muscular dystrophy in cultured human cardiomyocytes and a mouse model.
Lisa Boyette Started a Nonprofit to Save Her Brother and Cure Other Diseases
Alison F. Takemura | Oct 1, 2016
Founder and CEO, Curable; Adjunct Assistant Professor, University of Pittsburgh. Age: 35
Newly Discovered Hormone Explains Disease
Kerry Grens | Apr 15, 2016
Patients with neonatal progeroid syndrome lack a glucose-releasing hormone, while people with insulin resistance have an abundance.
Tanya Lewis | Apr 11, 2016
An analysis of the genomes of nearly 600,000 healthy individuals reveals a handful of people who appear resistant to certain genetic disorders.
Disease-Causing Mutations in Healthy People
Amanda B. Keener | Jun 8, 2015
A large-scale genome sequencing effort identifies mutations with disease-causing potential at higher rates than expected.
Spinoff Company to Develop Rare Disease Drugs
Molly Sharlach | Jan 7, 2015
Maryland-based Vtesse will work with the US National Institutes of Health to advance treatments for Niemann-Pick Type C disease and other lysosomal storage disorders.
Rare Disease to Inform Ebola Susceptibility?
Jef Akst | Nov 4, 2014
Parents of children with the fatal genetic disease Niemann-Pick Type C are taking an active role in research to understand how mutations associated with the disease may protect against Ebola.
Muscle Disease Gene Identified in Fish
Ed Yong | Jun 4, 2013
Scientists discover gene behind an inherited muscle disorder by studying zebrafish embryos.
New Stem Cells on the Block
Dan Cossins | May 15, 2013
By reprogramming human fibroblasts into pluripotent stem cells with somatic cell nuclear transfer, scientists have come up with a viable alternative to iPSCs.
Telomeres Affect Gene Expression
Ed Yong | May 5, 2013
As telomeres shorten with age, genes as far as 1,000 kilobases away could be affected, including one responsible for an inherited muscle disease.