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.
Cytotherapy
; 26(11): 1411-1420, 2024 Nov.
Article
in En
| MEDLINE
| ID: mdl-38970612
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 ß-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.
Key words
Full text:
1
Database:
MEDLINE
Main subject:
Hematopoietic Stem Cells
/
Genetic Therapy
/
Hematopoietic Stem Cell Transplantation
Limits:
Humans
Language:
En
Journal:
Cytotherapy
Journal subject:
TERAPEUTICA
Year:
2024
Type:
Article
Affiliation country:
United States