How is Gene Therapy Being Used to Treat Cancer and Other Diseases?
From the desk of Kumari Suninda
Gene therapy is a method of treating or preventing disease by altering the genetic code in a person’s cells.
When a gene is missing, or overactive, vital bodily functions can be impaired. One approach to gene therapy is to correct such problems and fix them at the source. This type of gene therapy, called in vivo gene therapy, uses viruses or gene editing tools in a patient’s body like CRISPR, to correct genetic abnormalities in specific cells. Dana-Farber’s Stuart Orkin, M.D., was the first to envision this genetic correction and the possibility of gene therapy.
How Do Gene Therapies Work?
In vivo gene therapies aim to use viral vectors or CRISPR gene editing tools for genetic manipulation. These tools can detect, activate, or eliminate genes. Genes can be altered in cancer cells or surrounding tissues. The goal is for the newly introduced gene to cause the cancer cells to die or to disrupt a process that is essential for their survival, such as providing nutrients to the cancer cells and surrounding tissues to prevent blood from forming tumors in the 19th century.
While the approach holds great promise, it presents many obstacles such as “gene silencing,” where transplanted genes are impermeable in animal studies, approaches to gene transfer has yielded promising results in the treatment of prostate, lung and intestinal tumors.
Various gene transfer strategies have been tested in clinical trials. These screenings include cancers of the blood, head and neck, liver, testes, prostate, bladder and other organs.
CAR T-cell therapy is currently approved for the treatment of:
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B-cell acute lymphoblastic leukemia
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a B-cell non-Hodgkin lymphoma
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Large B-cell lymphocytes
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Follicular lymphoma
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Mantle cell lymphoma
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Multiple myeloma
Efforts are underway to develop indirect CAR T-cell therapies derived from healthy donors that do not require the patient to harvest and engineer their own cells.
There are several CAR T-cell therapy trials for leukemia and solid tumors in adults and several recently opened trials for pediatric cancer.
View more info and detailed Table of Contents here: https://skyquestt.com/report/cancer-gene-therapy-market
How Does Cancer Gene Therapy Work?
Creating gene and cell therapies requires specialized skills. Procedures include genetic manipulation, collection of cells (usually from the patient), specialized cells, processing methods and packaging.
Dana-Farber's Connell & O'Reilly Families Cell Manipulation Core Facility (CMCF) plays a key role in advancing cell-gene therapy for cancer and prepares many modified and non-modified cell therapies for patients at Dana-Farber. CMCF is one of the largest academic cell therapy centers, with more than 70 members.
Current Status of Gene Therapy for the Treatment of Cancer
Gene therapy is the injection of foreign genomic material into the patient tissue to alter gene expression or cellular biology for therapeutic use. Initially, the primary goal of gene therapy was to deal with inherited diseases. Technological advances have facilitated gene therapy for the prevention and management of incurable diseases. Researchers are cautiously moving forward, hoping it will lead to safe and effective treatment for patients with single gene and complex inherited disorders.
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