In 1990, two seminal studies introduced a novel method, systematic evolution of ligands by exponential enrichment (SELEX), to discover and isolate…

Editor-in-Chief of Molecular Therapy, Dr. Joseph Glorioso, is joined by Rachael Nimmo, PhD, former director of the Cell Technology Group at Oxford Biomedica, and Kyriacos Mitrophanous, PhD, Chief Innovation Officer at Oxford Biomedica, to discuss an article recently published in Molecular Therapy by Nimmo and colleagues titled “Efficient in vivo generation of CAR T cells using a retargeted fourth-generation lentiviral vector.” Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au

The use of AAV, the gold-standard viral vector for gene therapy, has successfully progressed from preclinical studies to multiple FDA-approved therapies.…

The use of AAV, the gold-standard viral vector for gene therapy, has successfully progressed from preclinical studies to multiple FDA-approved therapies.…

The use of AAV, the gold-standard viral vector for gene therapy, has successfully progressed from preclinical studies to multiple FDA-approved therapies.…

Shi Xu and colleagues report a novel ionizable lipid-based LNP system that exhibits high mRNA delivery efficiency and develop a phylactic mRNA vaccine providing cross-protection against multiple serotypes of Streptococcus pneumoniae. This work advances the development of mRNA vaccine for combating bacterial infections.

From the first use of chemotherapy in the 1960s to the first FDA-approved CAR T-cell therapy in 2017, the treatment of pediatric cancers has evolved to become safer and more effective, increasing survival rates from 10% to nearly 80%. Despite these pivotal successes, cancer remains a leading cause of death among children and adolescents. This special issue is focused on the history, current state, and future of pediatric cancer therapy. We invite authors to submit research including the following treatment modalities of interest: Gene therapies Cell-based therapies Molecular therapies Viral therapies Precision oncology Vaccines Antibodies Immune checkpoint inhibitors Submission due date: September 30, 2025 This special issue will include invited reviews on the following topics: Current and emerging strategies for prevention of relapse post-stem cell transplant in children and young adults with acute leukemia Hema Rangarajan, MD, and Alex Rankin, MD, Nationwide Children's Hospital Cancer-associated fibroblasts (CAFs) in pediatric solid tumors Julie Tomolonis, MD, PhD, Elizabeth Garfinkle, PhD, Elaine Mardis, PhD, and Timothy Cripe, MD, PhD, Nationwide Children's Hospital Current landscape of CAR T-cell therapy in children and young adults with B-cell acute lymphoblastic leukemia Sara Silbert, MD, MHSc, Alexandra Dreyzin, MD, Flavia Mesquita Gava, MD, Chelsea O’Koren, MD, and Julie Ma, MD, National Cancer Institute Limitations of preclinical modeling in CAR T cells Victoria Giordani, MD, Julie Ma, MD, Josquin Moraly, MS, MD, Rosa Nguyen, MD, PhD, and Brynn Duncan, MD, National Cancer Institute Updates on cell therapy for AML Anand Bhagwat, MD, PhD, Children's Hospital of Philadelphia and Swati Naik, MBBS, St. Jude Children's Research Hospital Advances in immune-based therapies for pediatric bone sarcomas Brian Ladle, MD, PhD, and Nico Llosa, MD, Johns Hopkins University School of Medicine Advances in pediatric oncology: Challenges and opportunities in the low- and middle-income countries Chetan Dhamne, MD, Ramandeep Arora, MBBS, DCH, MRCPCH, CCT, MD, FRCPCH, Rahul Purwar, PhD, and Gaurav Narula, MD, Tata Memorial Hospital Adoptive cell therapies for CNS tumors: Current landscape and future directions Kun-Wei Song, MD, Aanchal Preet Kaur, PhD, and Sneha Ramakrishna, MD, Stanford School of Medicine Precision medicine in pediatric oncology: Current applications and future directions Nilay Shah, MD, Nationwide Children's Hospital and The Ohio State University College of Medicine Updates on rare pediatric cancer Jacquelyn Crane, MD, Children's Hospital of Philadelphia, Robyn Gartrell, MD, Johns Hopkins University School of Medicine, and Kris Ann Schultz, MD, Children's Minnesota, and Theodore W. Laetsch, MD, Children's Hospital of Philadelphia

This episode is the third in a series hosted by Molecular Therapy Editor-in-Chief Joseph Glorioso, PhD, that will cover comprehensive reviews of critical developments in the field of gene and cell therapy over the past 25 years.   In this episode, Dr. Glorioso will discuss the reviews, “Current trends in gene therapy to treat inherited disorders of the brain,” with author Beverly Davidson, PhD, The Children’s Hospital of Philadelphia, and “The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy,” with author Jeff Chamberlain, PhD, University of Washington School of Medicine.  If you enjoy this deep dive into research that is critical to the field, check out two more ASGCT events happening this fall: Advancing Cell and Gene Therapies for Cancer and Breakthroughs in Targeted In Vivo Gene Editing. During these events – in October and November, respectively – you’ll be able to hear directly from researchers about their own work related to these topics, or you can submit your own abstract for possible presentation. We hope to see you there! Music: 'Electric Dreams' by Scott Buckley - released under CC-BY 4.0. www.scottbuckley.com.au 

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