Following the discovery of nucleic acids by Friedrich Miescher in 1868, DNA and RNA were
recognized as the genetic code containing the necessary information for proper cell …

To date, almost 2600 gene therapy clinical trials have been completed, are ongoing or have
been approved worldwide. Our database brings together global information on gene therapy …

Mutations in the gene encoding dystrophin, a protein that maintains muscle integrity and
function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD …

Stem cells (SCs) maintain tissue homeostasis and repair wounds. Despite marked variation
in tissue architecture and regenerative demands, SCs often follow similar paradigms in …

Duchenne muscular dystrophy (DMD) is a monogenic disorder and a candidate for
therapeutic genome editing. There have been several recent reports of genome editing in …

Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by dystrophin gene
mutation. Conceptually, replacing the mutated gene with a normal one would cure the …

Viral vectors have been used for a broad spectrum of gene therapy for both acute and
chronic diseases. In the context of cancer gene therapy, viral vectors expressing anti-tumor …

Duchenne muscular dystrophy (DMD) is an X-linked, muscle wasting disease that affects 1
in 5000 males. Affected individuals become wheelchair bound by the age of twelve and …

Neuromuscular disorders comprise a diverse group of human inborn diseases that arise
from defects in the structure and/or function of the muscle tissue—encompassing the muscle …

Muscle stem cells, or satellite cells, are required for skeletal muscle maintenance, growth,
and repair. Following satellite cell activation, several factors drive asymmetric cell division to …