Molecular Therapy

A New Era of Molecular Therapy with Joseph Glorioso, PhD

Listen to the episode now. | November 06, 2024

This episode features a conversation between Dr. Roland Herzog and Dr. Joseph Glorioso.

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In Vivo Adenine Base Editing Rescues Adrenoleukodystrophy in a Humanized Mouse Model

Jessica Schneller, PhD | August 08, 2024

In this study, Gopalappa et al. sought to precisely correct the pathogenic p.G512S mutation, a frequently observed mutation in patients, using either an adenine base editor (ABE) or a prime editor (PE).

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AAV Capsid Production with Drs. Mario Mietzsch and Robert McKenna

Molecular Therapy Podcast | June 18, 2024

Listen today on the ASGCT Podcast Network!

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A New Vision for MTMCD with EiC Dr. Mohamed Abou-el-Enein

ASGCT Podcast Network | June 04, 2024

Mohamed Abou-el-Enein, MD, PhD, MSPH, the new editor-in-chief of Molecular Therapy Methods & Clinical Development (MTMCD), sits down with Scientific Editor Rory Bricker-Anthony, PhD, to discuss his vision for the journal.

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ASGCT Announces Joseph C. Glorioso, PhD, as Incoming Editor-in-Chief of Molecular Therapy

ASGCT & Molecular Therapy | May 09, 2024

Glorioso, a trailblazing gene therapy researcher and founding member of ASGCT, will assume the role on January 1, 2025.

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Prenatal Delivery of a Therapeutic Antisense Oligonucleotide Achieves Broad Biodistribution in the Brain and Ameliorates Angelman Syndrome Phenotype in Mice

Wan Du, MD, PhD | April 11, 2024

Read our monthly summary of a Molecular Therapy article from the April issue of The Vector.

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Hear Dr. Maxim Berezovski on the Molecular Therapy Podcast

Listen now! | February 13, 2024

Dr. Maxim Berezovski discusses a recent article published in Molecular Therapy Nucleic Acids.

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Hear Dr. Richard Vile on the Molecular Therapy Podcast

Listen now! | January 09, 2024

Dr. Vile speaks with Dr. Timothy Cripe about his recent article, "Trap and ambush therapy using sequential primary and tumor escape-selective oncolytic viruses."

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Mohamed Abou-el-Enein Appointed Editor of Molecular Therapy Methods and Clinical Development

Dr. Abou-el-Enein began five-year term Jan. 1. | January 04, 2024

The former associate editor-in-chief of MTMCD began his five-year term on Jan. 1.

ASGCT News  |  Molecular Therapy

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First Episode of Molecular Therapy Podcast Features Dr. Norbert Pardi

Listen now! | December 12, 2023

Listen to Dr. Pardi discuss his recent Molecular Therapy article, "Development of an mRNA-lipid Nanoparticle Vaccine Against Lyme Disease."

 

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Apply to Become Molecular Therapy's Next Editor-in-Chief

Send your CV + cover letter by Jan. 31, 2024. | October 17, 2023

Submit your CV and a cover letter by Jan. 31, 2024.

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Apply to Be the Next EIC of Molecular Therapy Methods & Clinical Development

Term Begins Jan. 2024 | June 30, 2023

The next editorial leader of MTMCD will assume the role on Jan. 1, 2024.

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

Courtney Bricker-Anthony, PhD | April 24, 2023

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.

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Improving the efficiency of homology directed repair in gene editing

Courtney Bricker-Anthony, PhD | March 24, 2023

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.

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Transcribing a New Approach to Angelman Syndrome

Courtney Bricker-Anthony, PhD | February 01, 2023

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.

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

Devin Rose | January 19, 2023

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

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