Molecular Therapy is the official journal of the American Society for Gene & Cell Therapy. Available in print and online, Molecular Therapy is dedicated to publishing important peer-reviewed research and cutting-edge reviews and promoting the sciences in genetics, medicine and biotechnology.

Publisher
Nature Publishing Group
Website
http://www.nature.com/mt/index.html

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New CRISPR base-editing technology slows ALS progression in mice

With a new CRISPR gene-editing methodology, scientists from the University of Illinois at Urbana-Champaign inactivated one of the genes responsible for an inherited form of amyotrophic lateral sclerosis—a debilitating and ...

Scientists improve DNA transfer in gene therapy

Parkinson's disease, Huntington's disease, cystic fibrosis and many other fatal human diseases are hereditary. Many cancers and cardiovascular diseases are also caused by genetic defects. Gene therapy is a promising possibility ...

Amniotic fluid yields alternatives to embryonic stem cells

Stem cells found in amniotic fluid can be transformed into a more versatile state similar to embryonic stem cells, according to a study published today in the journal Molecular Therapy. Scientists from Imperial College London ...