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.
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
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...
Gene and cell engineering advances have influenced almost all branches of life sciences. From the lab to the clinic, innovative gene synthesis technologies and gene editing tools have transf...
Inaugural Virtual Symposium: September 24 Register for our free online event: Advances in Drug Discovery and Development. On September 24, 2014, a world-class group of scientists from...
Gene and cell engineering advances have influenced almost all branches of life sciences. From the lab to the clinic, innovative gene synthesis technologies and gene editing tools have transf...
Inaugural Virtual Symposium: September 24 Register for our free online event: Advances in Drug Discovery and Development. On September 24, 2014, a world-class group of scientists from...