Emily Walsh Martin, PhD, spoke with Amber Freed, founder and CEO of SLC6A1 Connect, a patient organization focused on accelerating the development of therapies for SLC6A1 patients like her son Maxwell.

Emily Walsh Martin, PhD, spoke with Amber Freed, founder and CEO of SLC6A1 Connect, a patient organization focused on accelerating the development of therapies for SLC6A1 patients like her son Maxwell.

The American Society of Gene & Cell Therapy is the premier membership organization for professionals working in cell and gene therapy. On this network, we're sharing exciting stories from our members and the greater scientific community.

The American Society of Gene &amp; Cell Therapy is the premier membership organization for professionals working in cell and gene therapy. On this network, we're sharing exciting stories from our members and the greater scientific community.

ASGCT Podcast Network

Key Considerations of Gene Therapy Development: A Caregiver's Story

Dr. Anastasia Khvorova of UMass Chan Medical School joins The Issue Podcast to discuss the next frontier of siRNA therapeutics and delivery beyond the liver. During her discussion with co-hosts Sanjay D'Souza, PhD, and Jason West, PhD, she covers the kidney clearance barrier, local versus systemic delivery, how RNAi and gene editing complement each other, and where she expects the field to go over the next several years.

Dr. Anastasia Khvorova of UMass Chan Medical School joins The Issue Podcast to discuss the next frontier of siRNA therapeutics and delivery beyond the liver. During her discussion with co-hosts Sanjay D'Souza, PhD, and Jason West, PhD, she covers the kidney clearance barrier, local versus systemic delivery, how RNAi and gene editing complement each other, and where she expects the field to go over the next several years.

Beyond the Liver: Dr. Anastasia Khvorova on the Future of Extrahepatic siRNA Delivery

We are excited to announce that Dr. Paloma Giangrande has committed to a second term as Editor-in-Chief of Molecular Therapy Nucleic Acids (MTNA). In this episode, Dr. Marcin Kortylewski, the Oligonucleotides: Therapies and Applications section editor of MTNA, interviews Paloma about her journey into science and RNA biology, her perspectives on therapeutic development, her initiatives during her first term as Editor-in-Chief, and her vision for MTNA in the next five years. 

We are excited to announce that Dr. Paloma Giangrande has committed to a second term as Editor-in-Chief of Molecular Therapy Nucleic Acids (MTNA). In this episode, Dr. Marcin Kortylewski, the Oligonucleotides: Therapies and Applications section editor of MTNA, interviews Paloma about her journey into science and RNA biology, her perspectives on therapeutic development, her initiatives during her first term as Editor-in-Chief, and her vision for MTNA in the next five years.&nbsp;

The Future of Molecular Therapy Nucleic Acids

In this episode of Scientists, Unedited, Luigi Naldini, PhD, Professor of Cell and Tissue Biology and of Gene and Cell Therapy at the San Raffaele University School of Medicine and Scientific Director of the San Raffaele Telethon Institute for Gene Therapy, joins host Eirini Vamva, PhD, for a discussion on his story. Dr. Naldini, a pioneer of lentiviral vectors, reflects on the unexpected path that led him to gene therapy and helped turn a bold scientific idea into a clinical reality. He discusses the risks, setbacks, collaborations, and family support that shaped his career, as well as the future of gene therapy and why meaningful scientific progress requires persistence, teamwork, and time.

In this episode of Scientists, Unedited, Luigi Naldini, PhD, Professor of Cell and Tissue Biology and of Gene and Cell Therapy at the San Raffaele University School of Medicine and Scientific Director of the San Raffaele Telethon Institute for Gene Therapy, joins host Eirini Vamva, PhD, for a discussion on his story. Dr. Naldini, a pioneer of lentiviral vectors, reflects on the unexpected path that led him to gene therapy and helped turn a bold scientific idea into a clinical reality. He discusses the risks, setbacks, collaborations, and family support that shaped his career, as well as the future of gene therapy and why meaningful scientific progress requires persistence, teamwork, and time.

Key Considerations of Gene Therapy Development: A Caregiver's Story

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