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Muscular Dystrophy
Muscular Dystrophy is caused when atypical gene mutations impede the production of proteins essential to the formation of healthy muscle. The main symptoms of the group of diseases designated as muscular dystrophy are loss of muscle mass and weakness. Medication can be prescribed to reduce symptoms but there is no known cure.
SEP 30, 2020 | 12:00 AM
Cas9 based therapeutics have the potential to revolutionize the treatment of genetic diseases. However, safe and effective methods for delivering Cas9 protein, gRNA and donor DNA need to be...
JUL 02, 2020 | 1:30 PM
Genetic diseases are known occur due to various types of mutations in the human genome. The molecular assays to diagnose these diseases have been developed based on the type of mutation comm...
NOV 14, 2019 | 6:00 AM
Facioscapulohumeral muscular dystrophy (FSHD), one of the most common forms of progressive muscular dystrophies and is often conventionally diagnosed by Southern blot analysis. The accurate d...
JUL 01, 2017 | 12:00 AM
Disease-specific human pluripotent stem cells (hPSCs) represent a new chance to unravel cellular and molecular mechanisms of neurological diseases. Along this line, we were among the first to...
MAR 16, 2017 | 1:30 PM
Studying the genetics of rare congenital disorders disrupting cognitive function has led to the identification of multiple disease genes that helped us better understand the mechanisms underl...
AUG 30, 2016 | 8:00 AM
Over the last 15 years, human pluripotent stem cell (hPSC) technologies have progressed from academic curiosities into tools with the promise to underpin commerce, leading to real progress in...
SEP 24, 2014 | 6:00 AM
Duchenne Muscular Dystrophy (DMD) is an X-linked genetic disease occurring in 1 in 3,500 births, and is uniformly fatal in boys. It is caused by any one of over 50 known mutations in the gen...