Gene Therapy Trials Show Clinical Efficacy
Article Abstract:
Two gene therapy trials highlight the potential benefits of this treatment. A group of French scientists inserted a gene into stem cells that would correct X-linked severe combined immunodeficiency disease (SCID). The stem cells were intravenously infused into four babies with the disorder. In two babies, the stem cells matured into functioning T cells, which SCID patients lack. In another study, researchers inserted the gene for factor IX into a virus that could not reproduce. Three hemophilia patients were injected with the virus. Two of the patients no longer need to take as much factor IX, and take it less often.
Publication Name: JAMA, The Journal of the American Medical Association
Subject: Health
ISSN: 0098-7484
Year: 2000
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Researchers struggle with trials of stem-cell transplants for breast cancer
Article Abstract:
Physicians are finding it hard to enroll women in clinical trials of stem cell transplants in the treatment of breast cancer. These transplants of embryonic blood cells are used to boost the immune system, which can be damaged by chemotherapy. A clinical trial would evaluate the transplant against the standard treatment. But many women and their physicians are convinced that the transplants will extend life. For this reason, many women refuse to enter a clinical trial where they might not receive the transplant. One company has opened centers nationwide to provide women with transplants.
Publication Name: JAMA, The Journal of the American Medical Association
Subject: Health
ISSN: 0098-7484
Year: 1997
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New Therapies Show Promise for Patients With Leukemia, Hemophilia, and Heart Disease
Article Abstract:
Researchers discussed treatments for cancer, hemophilia, and heart disease at the annual meeting of the American Society of Hematology. A drug named Glivec has shown promise in treating chronic myelogenous leukemia. Hemophilia A can be treated with a non-viral gene therapy technique and heart failure can be treated with transplanted hematopoietic stem cells from bone marrow.
Publication Name: JAMA, The Journal of the American Medical Association
Subject: Health
ISSN: 0098-7484
Year: 2001
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