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Gene Therapy

Gene Therapy

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 inserted. The...

Gene Therapy

Gene Therapy

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 inserted. The...

Multipotency of Hematopoietic Stem Cells

Multipotency of Hematopoietic Stem Cells

The hematopoietic stem cells or HSCs are multipotent, meaning they can differentiate and give rise to all blood and immune cells. HSCs are maintained in the quiescent stage until an external stimulus initiates their differentiation. The multipotent HSCs exist as two heterogeneous populations, long-term repopulating cells (LTRC) and short-term repopulating cells (STRC). The two HSC populations have different surface markers or receptors and are classified based on quiescence and long-term...

Source And Potency Of Stem Cells

Source And Potency Of Stem Cells

Stem cells are undifferentiated cells with extensive self-renewal properties that help them maintain their population during the fetal and adult stages of life. They can specialize in all cell types of the human body. However, their differential potential may vary and can be classified into five types. Stem cells can be (1) Totipotent, (2) Pluripotent, (3) Multipotent, (4) Oligopotent, and (5) Unipotent. Each stem cell has a specific origin; the fertilized egg or zygote is a totipotent cell and...

Multipotency and Niche of Bulge Stem Cell

Multipotency and Niche of Bulge Stem Cell

A hair follicle or HF is a small part of the skin that produces the hair shaft. Paul Gerson Unna was the first to observe a bulge in the human hair follicle's outer root sheath (ORS). The bulge is present between the sebaceous gland and the arrector pili muscle and is the niche for hair follicle stem cells (HFSCs). The bulge is also a niche for melanocyte stem cells, and their loss results in graying of hair. The HFSCs express Sox9 and Lhx2, which help them maintain stemness and prevent...

Stem Cell Therapy for Tissue Regeneration

Stem Cell Therapy for Tissue Regeneration

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 that...

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Related Experiment Video

Updated: Jun 25, 2026

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

Published on: February 15, 2019

A 3D "nichoid" boost for gene-engineered blood stem cells.

Giulia Schiroli¹, Pietro Genovese²

¹Tessera Therapeutics, Somerville, MA, USA.

|February 6, 2026

Summary

This study introduces a nanoengineered substrate to support hematopoietic stem and progenitor cells (HSPCs) during gene therapy manufacturing. This approach reduces stress, enhances cell engraftment, and improves gene-edited cell output.

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Intrafemoral Injection of Human Hematopoietic Stem and Progenitor Cells into Immunocompromised Mice

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Last Updated: Jun 25, 2026

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

Published on: February 15, 2019

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications

Published on: August 9, 2022

Intrafemoral Injection of Human Hematopoietic Stem and Progenitor Cells into Immunocompromised Mice

Intrafemoral Injection of Human Hematopoietic Stem and Progenitor Cells into Immunocompromised Mice

Published on: December 8, 2023

Area of Science:

Biotechnology
Stem Cell Biology
Mechanobiology

Background:

Ex vivo manipulation of hematopoietic stem and progenitor cells (HSPCs) is crucial for gene therapies.
Current methods can cause culture-associated stress, impacting cell function and therapeutic efficacy.

Purpose of the Study:

To investigate the use of a nanoengineered 3D "nichoid" substrate for supporting HSPCs during ex vivo manipulation.
To determine if this substrate improves cell engraftment and polyclonal output after gene editing or lentiviral gene addition.

Main Methods:

Development and application of a nanoengineered 3D "nichoid" substrate.
Culture of CD34+ HSPCs on the nichoid substrate.
Assessment of cell engraftment and polyclonal output following gene editing or lentiviral gene addition.

Main Results:

The nichoid substrate mechanically supported HSPCs during ex vivo manipulation.
Reduced culture-associated stress was observed in cells cultured on the substrate.
Improved engraftment and polyclonal output were achieved after gene editing or lentiviral gene addition.

Conclusions:

Mechanobiology, through the use of the nichoid substrate, serves as a valuable manufacturing lever for enhancing HSPC gene therapies.
This approach holds promise for improving the efficacy and scalability of stem cell-based therapeutics.