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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Inhalable molecules neutralize SARS-CoV-2 in mice

Nanofitins, which are derived from a protein found in Sulfolobus acidocaldarius—an archaeal microorganism found in hot springs—successfully neutralized SARS-CoV-2 in mice and were well-tolerated. When inhaled by the rodents, ...

'Frameshifting' therapy for mast cell cancers reduces size, spread

A potential new treatment for mast cell cancers reduces the number of mast cells by "mutating" the messenger RNA (mRNA) before it can deliver instructions for manufacturing the gene responsible for cell proliferation. The ...

Direct-acting antiviral to treat COVID-19 developed in QLD

An international team of scientists from the Menzies Health Institute Queensland (MHIQ) at Griffith University and from City of Hope, a research and treatment center for cancer, diabetes and other life-threatening diseases ...

Making therapeutic sense of antisense oligonucleotides

Antisense oligonucleotides (ASO) hold great promise for pharmacotherapy. Now, researchers at Tokyo Medical and Dental University (TMDU) and Ionis Pharmaceuticals, advancing their earlier work on a heteroduplex oligonucleotide ...

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 ...

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