Related Experiment Video
Updated: May 12, 2025
08:32
CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
Published on: August 9, 2022
3.4K
Clinical hematopoietic stem cell-based gene therapy
Tami John1, Agnieszka Czechowicz1
1Department of Pediatrics, Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, and Center for Definitive and Curative Medicine, Stanford University School of Medicine, Stanford, CA 94305, USA.
Summary
Hematopoietic stem cell (HSC) gene therapy is revolutionizing treatment for genetic disorders. Advances in gene editing and viral vectors offer safer, more effective cures for patients worldwide.
Area of Science:
- Hematology
- Gene Therapy
- Genetics
Background:
- Hematopoietic stem cell (HSC) gene therapy has advanced significantly, transforming treatment for inherited diseases.
- Progress in viral vector technology and cell manufacturing has improved gene delivery safety and efficiency.
Purpose of the Study:
- To review the history and advancements in HSC gene therapies.
- To provide an overview of current clinical innovations and cell-therapy products.
- To discuss challenges and future opportunities in HSC gene therapy.
Main Methods:
- Review of historical data and advancements in viral vector technologies.
- Analysis of genome-editing techniques, including CRISPR-Cas.
- Examination of clinical trial outcomes and regulatory approvals.
Main Results:
- HSC gene therapies are increasingly approved and available globally.
- CRISPR-Cas technologies enable precise genome alterations for gene correction.
- These therapies offer long-lasting benefits with reduced toxicity compared to alternatives.
Conclusions:
- HSC gene therapy represents a paradigm shift in treating genetic disorders.
- Despite challenges, future opportunities aim for curative treatments through personalized medicine.
Related Concept Videos
00:59Gene Therapy
24.9K
Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
24.9K
01:21Stem Cell Therapy for Tissue Regeneration
4.0K
Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
Types of Stem Cells used in Stem Cell Therapy
The two main cell...
Types of Stem Cells used in Stem Cell Therapy
The two main cell...
4.0K
01:22Bone Marrow Sampling and Transplants
263
Bone marrow transplant is a potential cure for several diseases, including cancer and specific genetic disorders. Notably, this procedure is applicable for patients suffering from aplastic anemia, certain types of leukemia, severe combined immunodeficiency disease (SCID), Hodgkin's disease, non-Hodgkin's lymphoma, multiple myeloma, thalassemia, sickle-cell disease, and certain cancers.
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
263
01:01Regulation of Hematopoietic Stem Cells
3.1K
All blood and immune cells are produced from the multipotent hematopoietic stem cells (HSCs) by the process of hematopoiesis. However, they all have a limited life span. In addition, many are depleted in immune surveillance or combatting an injury or infection. This makes blood one of the most regenerative tissues. Hematopoiesis helps replenish these blood and immune cells, restoring the body's normal functioning. However, overproduction of blood and immune cells can make them cancerous or...
3.1K
01:22iPS Cell Differentiation
2.6K
The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
2.6K
01:17Stem Cell Culture
5.0K
Stem cell research aims to find ways to use stem cells to regenerate and repair cellular damage. Over time, most adult cells undergo the wear and tear of aging and lose their ability to divide and repair themselves. Stem cells do not display a particular morphology or function. Adult stem cells, which exist as a small subset of cells in most tissues, keep dividing and can differentiate into a number of specialized cells generally formed by that tissue. These cells enable the body to renew and...
5.0K