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Gene Therapy

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General public
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medical...
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UFMG

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documents in English
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  1. The concept of gene therapy
  2. The clinical spectrum
  3. Gene delivery strategies
  4. The delivery vector
  5. Retroviral vectors
  6. Adenoviral vectors
  7. Adeno associated virus
  8. Selected disease applications
    1. Cystic fibrosis
    2. Hemophilia
    3. Myocardial and peripheral ischemia
    4. HIV infection and aids
  9. Replication competent viruses
  10. Recommendations
  11. Bibliography

Gene therapy is an experimental form of treatment whereby sequences of nucleic acids (i.e., genes) are delivered to cells to change their biologic function. The concept initially arose as replacement therapy for monogenic inherited disorders. For these disorders, the aim is to replace a defective gene with the normal counterpart. The delivered genetic material undergoes transcription and translation using the host cell's machinery, leading to in situ production of the normal protein and thereby correction of the phenotypic defect.

[...] The only vector-related death in gene therapy trials to date occurred acutely following infusion of a high dose of adenovirus into the hepatic artery. Efforts to improve the cell specificity, safety, and efficacy of adenoviral vectors are being made by imparting targeting properties to the vector. These strategies involve genetic modification of the capsid proteins or the use of separate adapter molecules to attach cell-specific ligands and thereby avoid the virus's reliance on CAR. This is especially relevant for tumor therapy, because most tumors tend to have low CAR levels. [...]


[...] Thus, the effects of tumor suppressor gene therapy extend beyond the initial hypothesis in complex and often ill-defined manners. Despite these bystander effects, however, clinical trials of tumor suppressor genes have had disappointing efficacy, albeit with minimal toxicity. The basic issue of gene delivery efficiency remains a significant problem. MOLECULAR CHEMOTHERAPY In molecular chemotherapy the rationale is to achieve tumor expression of enzymes that convert nontoxic systemically administered prodrugs into their toxic counterparts, to achieve high tumor concentrations while avoiding systemic side effects. [...]


[...] Retroviral vectors for gene therapy are typically based on the Moloney murine leukemia virus (Mo-MLV). These viruses can carry transgenes of up to 8 kilobases in length. Generally, the envelope protein of these vectors are modified from the wild-type virus by replacing the Mo-MLV protein with that of either amphotropic virus 4070A or vesicular-stomatitis virus glycoprotein (VSVG), which greatly broadens the range of cells that can be infected. After infection, linear, double-stranded DNA is generated from the RNA viral genome by the viral enzyme reverse transcriptase. [...]

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