Related Experiment Video
Updated: Apr 25, 2026

07:04
Electroporation-Based Genetic Modification of Primary Human Pigment Epithelial Cells Using the Sleeping Beauty Transposon System
Published on: February 4, 2021
2.0K
My career path for developing gene therapy for blinding diseases: the importance of mentors, collaborators, and
1Department of Ophthalmology and Center for Advanced Retinal and Ophthalmic Therapeutics, Perelman School of Medicine, University of Pennsylvania , Philadelphia, PA 19104.
Human Gene Therapy
|August 20, 2014
Abstract
No abstract available in PubMed .
Related Concept Videos
Gene Therapy
22.1K
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...
22.1K
Gene Therapy
4.4K
4.4K
Blinding
2.9K
Blinding is a commonly used method of not telling participants which treatment a subject is receiving. Blinding is a critical part of a randomized control trial or RCT. It reduces the bias that affects the results. In an RCT, blinding is used in the form of a placebo. A placebo effect occurs when untreated subjects falsely believe they have received the treatment and report improved symptoms. A placebo or a dummy treatment is administered to subjects to negate the bias caused by such an effect.
2.9K
iPS Cell Differentiation
2.2K
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.
2.2K
Targeted Cancer Therapies
7.0K
The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
There are several types of targeted therapies against...
7.0K
Pharmacogenomics: Identification of New Drug Targets
119
Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
119

