Molecular Therapy

The Promise of Durable Remission in B-cell Malignancies With CAR-T Persistence

New research published in Molecular Therapy – Oncolytics shows a relationship between durable remission and the persistence of CAR-T cells in patients with B-cell malignancies.

Improving the efficiency of homology directed repair in gene editing

CRISPR/Cas9 has revolutionized the field of gene editing, but its efficiency in editing non-dividing, therapeutically relevant cells is limited by the induction of the error-prone non-homologous end joining repair pathway. Bak & colleagues developed an enrichment approach that promoted the efficiency of a more precise endogenous repair mechanism, homology directed repair (HDR), which led to greater numbers of successfully edited cells.

Transcribing a New Approach to Angelman Syndrome

Angelman syndrome is a severe neurogenetic disorder caused by the loss of UBE3A gene expression in the brain. In a recent Molecular Therapy paper, researchers showed how artificial transcription factors (ATFs) could be a promising approach to treating AS. Treated mice showed improvement in motor function, more activity in the open field test, and higher stride frequency than their untreated counterparts.

Meet New Editor-in-Chief of Molecular Therapy–Oncolytics: Dr. Tim Cripe

Dr. Cripe assumed his role with Molecular Therapy Oncolytics on Jan. 1, replacing Yuman Fong, MD.

Engineering Improved Neuroprotection in Glaucoma

Researchers used AAV-mediated gene therapy in the eyes to enhance neuroprotection in mouse models of glaucoma—the leading cause of irreversible blindness. Intraocular injections resulted in partial recovery of visual behavior.

Doubling Down on Mitochondrial dsRNAs in Sjӧgren’s Syndrome

From the Molecular Therapy family: Read a summary on mitochondrial double-stranded RNAs and watch our first-ever video abstract.

In Molecular Therapy: EBAG9 silencing exerts immune checkpoint function without adverse effects

Wirges et al demonstrate that EBAG9 downregulation in murine T cells and human CAR T cells augmented the cytolytic activity of these cells against hematopoietic tumors in vitro and in vivo.

Read Dr. Schneller's debut summary for The Vector!

Molecular Therapy Family's Impact Factor Increased 44 Percent in 2020

Molecular Therapy-Oncolytics' 75% increase, to 7.200, was the highest impact factor jump across more than 50 Cell Press journals.

Meet the Editor: Gerhard Bauer, Molecular Therapy—Methods & Clinical Development

ASGCT member and director of the Good Manufacturing Practice (GMP) laboratory at the UC Davis Institute for Regenerative Cures Gerhard Bauer will become the editor-in-chief of ASGCT's journal, Molecular Therapy—Methods & Clinical Development on January 1, 2020.

Meet the Editor: Roland W. Herzog, Ph.D., Editor-in-Chief of Molecular Therapy

ASGCT member and Indiana University professor of pediatrics Roland W. Herzog, Ph.D. will become the editor-in-chief of ASGCT's field-leading journal, Molecular Therapy, on January 1, 2020.

Molecular Therapy Family of Journals Welcomes Two Editors-in-Chief

Herzog and Bauer will assume their roles as editors-in-chief of ASGCT’s Molecular Therapy and Molecular Therapy—Methods & Clinical Development, respectively, on January 1.

A New AAV Vector Can Efficiently Target the Brains of Mice Through IV Injection

Researchers have developed this new tool for genetically modifying the brains of mice, and experiments in non-human primates are currently underway.

Molecular Therapy Impact Factors Jump Nearly 28 Percent Across Family of Titles

Parent journal, Molecular Therapy, now tops the field with a 19.9 percent jump in impact factor to 8.402.

ASGCT’s Launch Month Continues: Molecular Therapy App & 2018 Annual Meeting Website

Even before ASGCT launched a redesigned website last week, Society members were celebrating the release of a standalone app for the Molecular Therapy family of journals and unveiling the website for the 21st Annual Meeting.