Stem Cell Gene Therapy
Stem Cell Gene Therapy - Related Videos
08:32
CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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Cited by 6 •
2022
The present protocol describes an optimized hematopoietic stem and progenitor cell (HSPC) culture procedure for the robust engraftment of gene-edited cells in vivo.
06:59
Using the BLT Humanized Mouse as a Stem Cell based Gene Therapy Tumor Model
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Cited by 35 •
2012
The generation and characterization of tumor specific T cells using humanized mice is described here. Human thymic tissue and genetically modified human hematopoietic stem cells are transplanted into immunocompromised mice. This results in the reconstitution of an engineered human immune system allowing for in vivo examination of anti-tumor immune responses.
11:16
Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
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Cited by 9 •
2019
The goal of this protocol is to isolate nonhuman primate CD34+ cells from primed bone marrow, to gene-modify these cells with lentiviral vectors, and to prepare a product for infusion into the autologous host. The total protocol length is approximately 48 h.
22:06
Derivation of Hematopoietic Stem Cells from Murine Embryonic Stem Cells
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Cited by 7 •
2007
This protocol details the derivation of transplantable hematopoietic stem cells from mouse embryonic stem cells (ESC) and their subsequent injection into lethally irradiated recipient mice. Briefly, ESC are differentiated as embryoid bodies, which are then infected with retroviral HoxB4 and co-cultured with OP9 stromal cells and hematopoietic cytokines.
11:13
Adenoviral Gene Therapy for Diabetic Keratopathy: Effects on Wound Healing and Stem Cell Marker Expression in Human Organ-cultured Corneas and Limbal Epithelial Cells
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Cited by 27 •
2016
An example of adenoviral gene therapy in the human diabetic organ-cultured corneas is presented towards the normalization of delayed wound healing and markedly reduced epithelial stem cell marker expression in these corneas. It also describes the optimization of this process in stem cell-enriched limbal epithelial cultures.
FAQs
Self-renewal allows modified stem cells to persist after transplantation and maintain a continuing source of functional progeny. In hematopoietic applications, this persistence supports repeated production of healthy blood cells rather than a short-lived response from mature cells alone. Consequently, durable therapeutic benefit depends not only on introducing the genetic modification, but also on retaining modified cells that can repopulate the relevant tissue.
A therapeutic gene can supply a functional genetic instruction, while a gene-editing system can alter faulty genetic information. Depending on the disorder and strategy, modification may correct, replace, or regulate gene activity. These different approaches aim to restore tissue function through the cells’ descendants, making the choice of genetic intervention central to the expected biological outcome.
Gene expression determines whether modified cells produce the intended functional product, whereas engraftment determines whether those cells establish themselves successfully in the recipient. Effective treatment requires both: cells that carry a modification but fail to persist may provide limited benefit, while persistent cells with inadequate expression may not restore function. Safety risks must also be controlled throughout this process.
Before reinfusion, clinicians verify the modified stem cells as part of the ex vivo workflow. This step links the genetic intervention to the cell product that will be administered, rather than assuming that isolation and modification alone produced the intended result. Verification is therefore important for assessing whether the prepared cells are suitable for transplantation and for supporting controlled clinical use.
These disorders are particularly suited to a strategy using hematopoietic stem cells because the modified cells can repopulate the bone marrow and generate blood cells. If the genetic change remains functional in their progeny, newly produced cells may carry the corrected or regulated genetic activity. This connects the biology of blood-cell formation directly with the therapeutic goal of restoring systemic function.
The workflow begins by isolating a patient’s stem cells outside the body. Clinicians then use a vector to deliver either a therapeutic gene or a gene-editing system, verify the modified cells, and reinfuse the prepared population. The intended outcome is engraftment followed by production of functional progeny, with treatment success assessed through persistence, gene activity, and restored tissue function.