Gene therapy for blood diseases
Abstract
Genetic diseases affecting proteins and cells composing the blood may be treated by gene therapy using gene addition or gene editing methods. Protein deficiencies (e.g. hemophilia) are being approached using in vivo gene delivery by adeno-associated virus (AAV) vectors for therapeutic gene addition or gene editing. Blood cell diseases (e.g. sickle cell disease) are being approached using ex vivo gene addition or gene editing to treat isolated blood-forming hematopoietic stem cells or T cells that are then re-transplanted. In recent years, there has been much progress, and gene therapy is now routinely providing clinical benefit to patients with a variety of conditions. Several of these gene therapies have been licensed in the U.S. and EU and more for other disorders are being advanced toward licensure. The scope of therapeutic activity for gene therapy is expected to continue to expand as the technical capabilities advance.
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The post-mitotic nature of neurons prevents them from being harvested for ex vivo modification followed by re-infusion, as is commonly done for hematological disorders.
“Additionally, the post-mitotic nature of neurons prevents them from being harvested for ex vivo modification followed by re-infusion, as is commonly done for hematological disorders [79].”
From Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders (2022) · cites this paper as [79]
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“Over the past decade, ex vivo gene therapy has advanced, with several treatments approved for clinical use.(1) In vivo somatic cell gene therapy for treatment of blindness caused by mutations in the RPE65 gene is approved by Food and Drug Administration (FDA) and clinical trials for hereditary hemophilias are yielding…”
BackgroundCollaborative science to advance gene therapies in resource-limited parts of the world
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“…those involving deficiencies of plasma proteins, such as in hemophilia, and of other enzymes, such as lysosomal storage disease, and (b) those affecting blood cells, such as erythrocytes in hemoglobinopathies, leukocytes in immune disorders, and hematopoietic stem cells in Fanconi’s anemia [52].”
InfluentialImpact of molecular rigidity on the gene delivery efficiency of core-shell tecto dendrimers.
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- Authors
- Kohn, D. B.
- Affiliations
- University of California, Los Angeles (US)
- Corresponding author
- Kohn, Donald B
- ORCID
- Kohn, Donald B
- Venue
- Current Opinion in Biotechnology
- Publisher
- Elsevier BV
- Published
- 2019-12
- Volume
- 60
- Pages
- 39-45
- ISSN
- 0958-1669 (ISSN-L)
- OpenAlex
- W2947504994
- Cited by
- 39
- References
- 0
- Citations by year
- 2026: 2 · 2025: 4 · 2024: 4 · 2023: 4 · 2022: 4 · 2021: 10 · 2020: 7 · 2019: 3
- FWCI
- 0.69
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Current Opinion in Biotechnology (publishedVersion)
- PubMed (publishedVersion)
- Funders
- National Institutes of Health
- California Institute for Regenerative Medicine
- Doris Duke Charitable Foundation
- Broad Stem Cell Research Center
- BioMarin Pharmaceutical Inc.
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Hematology › Hemophilia Treatment and Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- gene therapy, hematologic disorders, hemophilia, sickle cell disease, adeno-associated virus vectors, genome editing, hematopoietic stem cells, T cells, ex vivo gene therapy, in vivo gene delivery, gene addition, protein deficiency
- MeSH terms
- Gene Editing, Dependovirus, Genetic Vectors, Hematologic Diseases, Humans, Genetic Therapy
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