International Society for Cell & Gene Therapy Stem Cell Engineering Committee report on the current state of hematopoietic stem and progenitor cell-based genomic therapies and the challenges faced

Ashish O. Gupta, Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA.
Melissa Azul, Division of Hematology and Oncology, Department of Pediatrics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
Senthil Velan Bhoopalan, Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
Allistair Abraham, Department of Pediatrics, The George Washington University School of Medicine and Health Sciences, Washington, DC, USA.
Alice Bertaina, Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University, Stanford, California, USA.
Alan Bidgoli, Division of Blood and Marrow Transplantation, Children's Healthcare of Atlanta, Aflac Blood and Cancer Disorders Center, Emory University, Atlanta, Georgia, USA.
Carmem Bonfim, Pediatric Blood and Marrow Transplantation Division and Pelé Pequeno Príncipe Research Institute, Hospital Pequeno Príncipe, Curitiba, Brazil.
Amy DeZern, Bone Marrow Failure and MDS Program, Johns Hopkins Medicine, Baltimore, Maryland, USA.
Jingjing Li, Graduate School of Biomedical Engineering, University of New South Wales, Sydney, New South Wales, Australia.
Chrystal U. Louis, TScan Therapeutics, Waltham, Massachusetts, USA.
Duncan Purtill, Department of Haematology, Fiona Stanley Hospital, Perth, Western Australia, Australia.
Annalisa Ruggeri, IRCCS Ospedale San Raffaele, Segrate, Milan, Italy.
Jaap Jan Boelens, Stem Cell Transplantation and Cellular Therapies, Memorial Sloan Kettering Cancer Center, New York, New York, USA.
Susan Prockop, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, Massachusetts USA.
Akshay Sharma, Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, Tennessee, USA. Electronic address: akshay.sharma@stjude.org.

Document Type

Journal Article

Publication Date

6-6-2024

Journal

Cytotherapy

DOI

10.1016/j.jcyt.2024.06.002

Keywords

adrenoleukodystrophy; gene editing; gene therapy; hematopoietic stem cells; metachromatic leukodystrophy; sickle cell disease; thalassemia

Abstract

Genetic manipulation of hematopoietic stem cells (HSCs) is being developed as a therapeutic strategy for several inherited disorders. This field is rapidly evolving with several novel tools and techniques being employed to achieve desired genetic changes. While commercial products are now available for sickle cell disease, transfusion-dependent β-thalassemia, metachromatic leukodystrophy and adrenoleukodystrophy, several challenges remain in patient selection, HSC mobilization and collection, genetic manipulation of stem cells, conditioning, hematologic recovery and post-transplant complications, financial issues, equity of access and institutional and global preparedness. In this report, we explore the current state of development of these therapies and provide a comprehensive assessment of the challenges these therapies face as well as potential solutions.

Department

Pediatrics

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