The CMT Research Foundation is launching a new Journal Club webinar series designed specifically for researchers working in the CMT space. Each session will spotlight a recent, high-impact publication, with a short presentation from the research team followed by open discussion and Q&A. It is a low-lift way to stay current on the field, hear directly from the scientists doing the work, and connect with peers who are tackling similar problems. 

Session One: July 30, 2026, 11:00 a.m. ET 

The first session will focus on a recent paper published in Molecular Therapy: Nucleic Acids, “Safety, efficacy, and distal nerve Schwann cell biodistribution in non-human primates and rodent models to support translation of AAV9 RNAi therapy for CMT1A.” 

You can register for the discussion here. 

CMT1A, the most common inherited form of Charcot-Marie-Tooth disease, is caused by a duplication of the PMP22 gene, which leads to overproduction of PMP22 protein in Schwann cells and disrupts the myelin that protects peripheral nerves. This study describes a gene silencing approach that uses an AAV9 vector to deliver a therapeutic microRNA (miR871) directly targeting PMP22 mRNA, administered via a single lumbar intrathecal injection. 

The research team’s findings include: 

• Confirmed on-target specificity of miR871 for PMP22 in vitro 

• Identification of a safe and effective dosing range in rodent models 

• No significant toxicity observed in rodent or non-human primate studies 

• Evidence that the AAV9 vector successfully reaches Schwann cells in distal peripheral nerves, a critical hurdle given the much greater nerve length in large animals and humans compared to smaller research models 

• PMP22 target engagement and protein-level silencing in both rodent and non-human primate models, at levels expected to support normal myelination in humans 

Together, these results help lay the groundwork for translating this RNAi approach toward a human clinical trial for CMT1A, a milestone many in the CMT research and patient communities have been anticipating for years. 

What to Expect 

This session will feature a short, formal presentation of approximately 20 to 30 minutes from the research team, covering the study’s design, key findings, and translational implications. The presentation will be followed by an open discussion and Q&A, giving attendees the chance to ask questions directly, dig into methodology, and discuss how these findings might inform their own work. 

Why Attend 

• Get the findings straight from the source. Skip the abstract and hear directly from the researchers about what the data show and what it means for the field. 

• Ask your questions live. The Q&A format is built for genuine scientific dialogue, not just a passive webinar. 

• Stay connected to the CMT research community. These sessions are a chance to see what your peers are working on and where the field is heading. 

• No travel required. Get convention-quality scientific content without leaving your desk. 

Who Should Join 

This series is built for researchers, clinicians, and trainees actively working in or adjacent to the CMT field, including those focused on foundational biology, preclinical research, drug discovery, and clinical translation. 

Save Your Seat 

Registration is now open for the first session on July 30 at 11:00 a.m. ET. Save your spot below. 

Register Here 

Questions about the Journal Club series can be directed to the CMT Research Foundation’s Kate Andersh via [email protected].