genetherapy-deepwater：基因療法-深水(編輯修改稿)
2025-02-08 08:03 本頁面
【文章內(nèi)容簡(jiǎn)介】 experimenters in this field. First Human Gene Therapy attempt ? Began in 1990, using ex vivo strategy, where patient cells were cultivated in laboratory and incubated with vectors ? transplanted back into the patient ? Attempted to treat 2 geic disorders, including children with immune defiency as well as people with high levels of serum cholesterol First Human GT cont… ? Practical approaches, delivering genes based on socalled in vivo GT where viruses are directly given to patients ? First model was dependent on a version of adenovirus to treat cystic fibrosis GT on Cancer Treatment ? Researchers are working to improve the body39。s natural ability to fight the disease or to make the cancer cells more sensitive to other kinds of treatment, such as chemotherapy ? Substitution of a working copy of a gene for an inactive or defective gene. GT on Cancer cont… ? Injection of cancer cells with a gene that makes them more sensitive to treatment with an anticancer drug. ? Introduction of the multidrug resistance gene into stem cells. The MDR gene is used to make the stem cells more resistant to the side effects of the high doses of anticancer drugs. Hurdles Summary ? There are many obstacles that prevent researchers and scientist from developing successful gene therapy techniques. ? The issues being confronted are the same ones that are faced whenever a powerful new technology is developed. When All is Said… ? A proposed gene therapy trial, or protocol, must be approved by at least two review boards at the scientists39。 institution. ? Any studies involving humans must be reviewed with great care. CASE STUDY: SCID ? SCID = Severe Combined Immunodeficiency What is SCID? ? SCID patients can neither – Construct cellmediated immune response –Make Antibodies ? Patients do not die of SCID itself ? More Boys than Girls SCID Cases, Past amp。 Present ? Not discovered until early 1960s ? Occur in 1 out of ,000 ? “Bubble Boy” – David Vetter Spent 12 years in Texas Hospital Sterile Tent ? Usually not discovered until too late Science Behind SCID (1) What Happens in SCID ? Lymphocyte T amp。 B cells do not get developed – Normal catabolism of p
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