Members of the Working Group on Pediatric Gene Therapy and Medical Ethics at NYU Langone have published research articles and delivered presentations about ethical issues surrounding gene-based therapies in pediatric populations, including sibling and discordant eligibility, expanded access policies, informed consent, and more. Below is a selection of our contributions to peer-reviewed academic journals and other reputable media outlets.

Peer-Reviewed Publications

Bateman-House A … Hunt C. Somatic gene therapy research in pediatric populations: Ethical issues and guidance for operationalizing early phase trials. Pharmaceut Med. 2023. DOI.

Shah LD … Bateman-House A. Fetal therapies and clinical research: Beyond risk and benefit. Am J Bioeth. 2022. DOI.

Kearns L … Bateman-House A. Gene therapy companies have an ethical obligation to develop expanded access policies. Mol Ther. 2021. DOI.

Webb J, Shah LD, and Bateman-House A. Siblings and discordant eligibility for gene therapy research: Considering parental requests for non-trial ‘compassionate use.’ Clinical Ethics. 2021. DOI.

Articles and Opinion Pieces

Hunt C, et al. Pediatric Gene Therapy Development Can Benefit From Trial Decentralization. Health Affairs Forefront. September 20, 2022.

Hunt C, Bateman-House A, and Shah LD. Uncertainties in pediatric gene therapy: Takeaways from a lunchtime lecture series. American Society of Gene and Cell Therapy Blog. March 4, 2022.

Hunt C and Kearns L. Ethical issues in pediatric gene therapy trials. Columbia University’s Center for ELSI Resources and Analysis Newsletter. January 25, 2021.

Webb J and Bateman-House A. It’s time to make ClinicalTrials.gov a better tool for patients. Here’s how. STAT First Opinion. April 28, 2020.

Shah L. New working group to produce guidance for pediatric gene therapy: Mission to advance research policy. Relias Media. April 1, 2020.

Video Lectures, Webinars, and Other Events

Operationalizing Informed Consent in Pediatric Gene Therapy Research (VIDEO), featuring Sandy Macrae, PhD, MB, ChB, president and CEO of Sangamo Therapeutics, Inc., with a response by Arthur L. Caplan, PhD, founding director of the Division of Medical Ethics at NYU Langone’s Department of Population Health. December 4, 2020.

Lived Experiences: The Personal and Family Impact of Participating in Pediatric Gene Therapy Research (VIDEO), featuring John F. Crowley, JD, MBA, chairman and CEO of Amicus Therapeutics, with a response by Patrick Moeschen, MA, teacher and muscular dystrophy patient advocate. December 3, 2020.

Ethical Challenges of Immunogenicity and Toxicity in Pediatric Gene Therapy Research (VIDEO), featuring James Wilson, MD, PhD, Rose H. Weiss Professor and director of the Orphan Disease Center and Gene Therapy Program at the University of Pennsylvania Perelman School of Medicine, with a response by Timothy Cripe, MD, PhD, chief of pediatric hematology and oncology and blood and marrow transplantation at Nationwide Children's Hospital. December 2, 2020.

Equity Issues in Pediatric Gene Therapy Research (VIDEO), featuring Pilar Ossorio, PhD, JD, professor of law and bioethics at University of Wisconsin Law School, with a response by Rafael Escandon, PhD, DrPH, MPH, senior vice-president of medical affairs, policy, and patient engagement at BridgeBio Pharma, Inc. December 1, 2020.

Risks and Benefits in Pediatric Gene Therapy Research (VIDEO), featuring Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, with a response by Pat Furlong, MS, RN, BSN, founder and president of Parent Project Muscular Dystrophy. November 30, 2020.