Session Details

Session Details

01:30 PM - 03:15 PM - Room 309-310

Vinod Prasad - Professor - Duke University Medical Center

Co-Chair

01:30 PM - 03:15 PM - Room 309-310

Brian Bigger - PhD - University of Edinburgh

Co-Chair

01:30 PM - 01:45 PM - Room 309-310

Maria Ester Bernardo - MD, PhD - San Raffaele Telethon Institute for Gene Therapy

46: Hematopoietic Stem Cell Gene Therapy for Hurler Syndrome: Interim Skeletal Outcome and Skeletal Cross-Correction Mechanisms

01:45 PM - 02:00 PM - Room 309-310

Shunji Tomatsu - PhD, MD - Nemours/Alfred I. DuPont Hospital for Children

47: Adeno-Associated Virus-Based Gene Therapy Delivering Combinations of Two Growth-Associated Genes

02:00 PM - 02:15 PM - Room 309-310

Laura Pisani - REGENXBIO Inc.

48: CAMPSIITE^™ Phase I/II/III: An Interim Clinical Study Update of RGX-121, an Investigational Gene Therapy for the Treatment of Neuronopathic Mucopolysaccharidosis Type II (MPS II)

02:15 PM - 02:30 PM - Room 309-310

Marine Laurent - UMRS_951 Genethon

49: Rescue of Lysosomal Acid Lipase Deficiency in Mice by AAV Liver Gene Transfer

02:30 PM - 02:45 PM - Room 309-310

Luis Tecedor - PhD - Children's Hospital of Philadelphia

50: AAV-Ep+ in Preclinical Studies for Late Infantile Neuronal Ceroid Lipofuscinosis

02:45 PM - 03:00 PM - Room 309-310

Brian Bigger - PhD - University of Edinburgh

51: Long-Term Haematopoietic Stem Cell Gene Therapy in Mucopolysaccharidosis IIIB Mice Corrects Disease with No Evidence of Insertional Mutagenesis Despite High Vector Copy Numbers

03:00 PM - 03:15 PM - Room 309-310

Jillian Gallagher - BS - UMass Chan Medical School

52: From Mouse to Sheep: Developing an AAV Gene Therapy for Sialidosis