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 Journal Article
| Authors: | Gupta, A. O.; Azul, M.; Bhoopalan, S. V.; Abraham, A.; Bertaina, A.; Bidgoli, A.; Bonfim, C.; DeZern, A.; Li, J.; Louis, C. U.; Purtill, D.; Ruggeri, A.; Boelens, J. J.; Prockop, S.; Sharma, A. |
| Article Title: | 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 |
| 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. © 2024 International Society for Cell & Gene Therapy |
| Keywords: | genetics; patient selection; cytology; metabolism; stem cell transplantation; hematopoietic stem cell transplantation; food and drug administration; genetic manipulation; stem cell; gene therapy; thalassemia; beta thalassemia; beta-thalassemia; hematopoietic stem cells; genomics; hematopoietic stem cell; biological therapy; therapy; cost benefit analysis; sickle cell disease; long terminal repeat; adrenoleukodystrophy; bone marrow depression; sickle cell anemia; anemia, sickle cell; procedures; cell engineering; metachromatic leukodystrophy; genetic therapy; humans; human; article; cell- and tissue-based therapy; gene editing; atidarsagene autotemcel; betibeglogene autotemcel; elivaldogene autotemcel; exagamglogene autotemcel; lenmeldy; lovotibeglogene autotemcel; lyfgenia; peroxisomal disorder; transplant complication |
| Journal Title: | Cytotherapy |
| Volume: | 26 |
| Issue: | 11 |
| ISSN: | 1465-3249 |
| Publisher: | Elsevier Science Ltd. |
| Date Published: | 2024-11-01 |
| Start Page: | 1411 |
| End Page: | 1420 |
| Language: | English |
| DOI: | 10.1016/j.jcyt.2024.06.002 |
| PUBMED: | 38970612 |
| PROVIDER: | scopus |
| PMCID: | PMC11471386 |
| DOI/URL: | |
| Notes: | Article -- Source: Scopus |
