Developing gene and cell therapies for rare diseases: an opportunity for synergy between academia and industry

Promising gene therapies pose million-dollar conundrum

(2016) Erika Check Hayden   NATURE

Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome

(2015) Salima Hacein-Bey Abina et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Gene therapy returns to centre stage

(2015) Luigi Naldini   NATURE

Gene Therapy Prolongs Survival and Restores Function in Murine and Canine Models of Myotubular Myopathy

(2014) M. K. Childers et al.   Science Translational Medicine

Mechanisms of Retroviral Integration and Mutagenesis

(2013) Alessia Cavazza et al.   HUMAN GENE THERAPY

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

(2013) A. Aiuti et al.   SCIENCE

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

(2013) A. Biffi et al.   SCIENCE

Lessons Learned from the Clinical Development and Market Authorization of Glybera

(2013) Laura M. Bryant et al.   Human Gene Therapy Clinical Development

Orphan drug development: an economically viable strategy for biopharma R&D

(2012) Kiran N. Meekings et al.   DRUG DISCOVERY TODAY

Limbal Stem-Cell Therapy and Long-Term Corneal Regeneration

(2010) Paolo Rama et al.   NEW ENGLAND JOURNAL OF MEDICINE

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

(2009) Alessandro Aiuti et al.   NEW ENGLAND JOURNAL OF MEDICINE