title: Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.
abstract: Survival rates after allogeneic hematopoietic cell transplantation (HCT) for Fanconi anemia (FA) have increased dramatically since 2000. However, the use of autologous stem cell gene therapy, whereby the patient's own blood stem cells are modified to express the wild-type gene product, could potentially avoid the early and late complications of allogeneic HCT. Over the last decades, gene therapy has experienced a high degree of optimism interrupted by periods of diminished expectation. Optimism stems from recent examples of successful gene correction in several congenital immunodeficiencies, whereas diminished expectations come from the realization that gene therapy will not be free of side effects. The goal of the 1st International Fanconi Anemia Gene Therapy Working Group Meeting was to determine the optimal strategy for moving stem cell gene therapy into clinical trials for individuals with FA. To this end, key investigators examined vector design, transduction method, criteria for large-scale clinical-grade vector manufacture, hematopoietic cell preparation, and eligibility criteria for FA patients most likely to benefit. The report summarizes the roadmap for the development of gene therapy for FA.
authors.affiliation: Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, Minnesota 55455, USA. tolar003@umn.edu
authors.forename: Jakub
authors.initials: J
authors.lastname: Tolar
authors.forename: Jennifer E
authors.initials: JE
authors.lastname: Adair
authors.forename: Michael
authors.initials: M
authors.lastname: Antoniou
authors.forename: Cynthia C
authors.initials: CC
authors.lastname: Bartholomae
authors.forename: Pamela S
authors.initials: PS
authors.lastname: Becker
authors.forename: Bruce R
authors.initials: BR
authors.lastname: Blazar
authors.forename: Juan
authors.initials: J
authors.lastname: Bueren
authors.forename: Thomas
authors.initials: T
authors.lastname: Carroll
authors.forename: Marina
authors.initials: M
authors.lastname: Cavazzana-Calvo
authors.forename: D Wade
authors.initials: DW
authors.lastname: Clapp
authors.forename: Robert
authors.initials: R
authors.lastname: Dalgleish
authors.forename: Anne
authors.initials: A
authors.lastname: Galy
authors.forename: H Bobby
authors.initials: HB
authors.lastname: Gaspar
authors.forename: Helmut
authors.initials: H
authors.lastname: Hanenberg
authors.forename: Christof
authors.initials: C
authors.lastname: Von Kalle
authors.forename: Hans-Peter
authors.initials: HP
authors.lastname: Kiem
authors.forename: Dirk
authors.initials: D
authors.lastname: Lindeman
authors.forename: Luigi
authors.initials: L
authors.lastname: Naldini
authors.forename: Susana
authors.initials: S
authors.lastname: Navarro
authors.forename: Raffaele
authors.initials: R
authors.lastname: Renella
authors.forename: Paula
authors.initials: P
authors.lastname: Rio
authors.forename: Julián
authors.initials: J
authors.lastname: Sevilla
authors.forename: Manfred
authors.initials: M
authors.lastname: Schmidt
authors.forename: Els
authors.initials: E
authors.lastname: Verhoeyen
authors.forename: John E
authors.initials: JE
authors.lastname: Wagner
authors.forename: David A
authors.initials: DA
authors.lastname: Williams
authors.forename: Adrian J
authors.initials: AJ
authors.lastname: Thrasher
mesh_terms: D003226:Congresses as Topic; D005199:Fanconi Anemia / Q000628:therapy*; D015316:Genetic Therapy / Q000379:methods*; D006412:Hematopoietic Stem Cells / Q000166:cytology; D006801:Humans; D033581:Stem Cell Transplantation / Q000379:methods