Lysosomal Disorders

Novel Film Matrix May Solve AAV-Based Therapy Production, Storage Challenges

Maria A. Croyle, PhD, discusses the development of a novel film matrix that can safely and effectively store and transport AAV-based gene therapies.

Addressing Challenges in Gene Therapy Vector Manufacturing and Storage

Maria A. Croyle, PhD, discusses the manufacturing challenges facing the gene therapy space and her inspiration for developing a novel preservation method for viral vector-based therapies.

Lentiviral-Based Gene Therapy for LAD-I Improves Clinical Course, CD18 Expression

Typically treated with stem cell transplantation, this lentiviral vector-based gene therapy approach may resolve challenges related to donor availability and post-transplantation complications.