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jscanlon95 jscanlon95
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9 years ago
Provide examples of major barriers that need to be addressed if gene therapy is to become a safe and reliable treatment for genetic diseases.
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wrote...
9 years ago
Gene therapy reflects major challenges in modern medicine and treatment technique as its too hard to introduce a gene of interest into the cells of the body and maintain them to working accurately.

Few eg of challenges are;

Insufficient precision of the targetting system- during gene therapy trial in treatment of children with X-linked Severe Combined Immune Deficiency (SCID)
Cost effectiveness of Gene Therapy- many gene therapy approaches are individualised and proove to be very cost effective. For eg., in case of cancer treatment, gene therapy could be very promising approach but it is highly cost effective.
The adverse immunological reactions to vector- It is possible that the vector used may lead to activation of immunological reponses against it. An eg is Jesse Gelsinger, had a rare liver-disorder, took part in gene therapy trial in his treatment in US.He died because of the complications from inflamatory response shortly after receiving a amall dose of experiemntal Adenovirus Vector. Immediatly the trial was stopped after his death.
The ability of the vector to revert into infectious virus- The virus used as vector may revert back as infectious virus and may cause the disease or increases its effects by activating immunological responses, leading to death.
wrote...
9 years ago
The insufficient precision of the targeting system.
The insufficient vehicle capacity of viral vectors
The adverse immunological reactions to vectors
The ability of the vector to revert to an infectious virus
The transience of gene therapy effects
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