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Category Archives: Genetic Therapy

Genetic Test Results: What to Do if You’ve Tested Positive

If you test positive foran abnormal BRCA1, BRCA2, or PALB2 gene and you have never had breast cancer, you now know that you are at much higher-than-average risk of developing it over the course of your lifetime. The average lifetime risk of breast cancer for women is about 12%. For women who have aBRCA1 or BRCA2 abnormality, the risk of developing breast cancer in your lifetime is between about 40% and 85% about 3 to 7 times greater than that of a woman who does not have the mutation. Continue reading

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Genome Editing for Hemophilia: A Next Step in Genetic …

Using an innovative gene therapy technique called genome editing that hones in on the precise location of mutated DNA, scientists have treated hemophilia, a blood clotting disorder, in mice. This is the first time that genome editing, which precisely targets and repairs a genetic defect, has been done in a living animal and achieved clinically meaningful results Continue reading

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Antisense therapy – Wikipedia

Antisense therapy is a form of treatment for genetic disorders or infections. When the genetic sequence of a particular gene is known to be causative of a particular disease, it is possible to synthesize a strand of nucleic acid (DNA, RNA or a chemical analogue) that will bind to the messenger RNA (mRNA) produced by that gene and inactivate it, effectively turning that gene “off” Continue reading

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Gene therapy – Mayo Clinic

Overview Gene therapy involves altering the genes inside your body’s cells in an effort to treat or stop disease. Continue reading

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Gene therapy | Cancer in general | Cancer Research UK

Gene therapy is a cancer treatment that is still in the early stages of research. Genes are coded messages that tell cells how to make proteins. Proteins are the molecules that control the way cells behave. Continue reading

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Pediatric MATCH: Targeted Therapy Directed by Genetic …

Experimental: Subprotocol A (NTRK1, NTRK2, or NTRK3 gene fusion) Patients with a NTRK1, NTRK2, or NTRK3 gene fusion receive Trk inhibitor LOXO-101 PO or via nasogastric- or gastric-tube BID on days 1-28. Courses repeat every 28 days for 2 years in the absence of disease progression or unacceptable toxicity. Continue reading

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