Related Experiment Video
Updated: May 16, 2026
06:10
Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates
Published on: May 9, 2025
Convergence of gene and cell therapy
Alexey Bersenev1, Bruce L Levine
1Department of Pathology and Laboratory Medicine, The University of Pennsylvania Perelman School of Medicine, Philadelphia, PA 19104, USA.
Regenerative Medicine
|December 6, 2012
Summary
Gene and cell therapies are advancing, with gene-modified cells enabling new treatments. Recent clinical developments show promise for future applications in regenerative medicine and disease treatment.
Area of Science:
- Biotechnology
- Cellular Engineering
- Gene Therapy
Background:
- Gene therapy and cell therapy have experienced fluctuating progress and public perception over the last 20 years.
- Recent advancements suggest a turning point with increasing successes in the field.
Purpose of the Study:
- To review the progress and emerging trends in gene and cell therapy.
- To highlight the role of gene-modified cells in therapeutic applications.
- To discuss future clinical potential based on recent developments.
Main Methods:
- Review of recent clinical developments in cellular engineering via gene transfer.
- Analysis of the applications of gene-modified cells in therapy.
- Synthesis of emerging trends and future prospects.
Main Results:
- Gene-modified cells offer novel strategies for engineering cellular properties.
- Cells can be engineered as effective vehicles for gene delivery.
- Methods for tracking cells and controlling their persistence post-transplantation are advancing.
Conclusions:
- The field of gene and cell therapy is gaining significant momentum.
- Gene-modified cells are crucial for current and future therapeutic innovations.
- Recent clinical successes pave the way for expanded future applications.
Related Concept Videos
00:59Gene 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...
00:59Gene 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...
01:24Methods of Nuclear Reprogramming
Nuclear reprogramming is a process of transforming one cell type into an unrelated cell type by epigenetic changes that alter the cell’s original gene expression pattern. Such epigenetic changes force cells to express a different set of genes, which play a significant role in inducing transformation into other cell types. Nuclear reprogramming offers applications in reproductive cloning for livestock propagation and regenerative medicine — developing patient-specific cells for injury repair.
01:21Stem 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...
Types of Stem Cells used in Stem Cell Therapy
The two main cell types that...
01:28Forced Transdifferentiation
Transdifferentiation, also known as lineage reprogramming, was first discovered by Selman and Kafatos in 1974 in silkmoths. They observed that the moths’ cuticle-producing cells transformed into salt-producing cells. Many such cases of natural transdifferentiation occur in organisms. In humans, pancreatic alpha cells can become beta cells. In newts, the loss of the eye’s lens causes the pigmented epithelial cells to transdifferentiate into the lens cells.
Artificial transdifferentiation occurs...
Artificial transdifferentiation occurs...
02:50Combination Therapies and Personalized Medicine
Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...