Human Gene Therapy - The Term Paper

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Of this there are two types:

Splicsome mediated - the delivery of RNA (a Repair Method) a. Strand that pair specifically with the intron next to the mutated segment of mRNA and leaves the correct strand.

b. Simultaneous delivery of a correct version of the segment to replace the mutated piece in the final RNA product.

Translation of the repaired mRNA to produce the normal functional protein.

Triple Helix-Forming Oligonucleotide: Oligonucleotides that bind the double strand of the mutated gene's DNA and actually forms a triple strand that blocks the mutation.

Antisense Technology is used to turn off a mutated gene in a cell by targeting the mRNA transcripts copied from the gene according to Crabtree. Further discussed in the role of Ribozymes (RNA enzymes). It is possible to have delivery of RNA strand that has been engineered to function as ribozymes to the cell, the RNA is encoded by a mutated gene preventing it from being translated into protein.

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Small interfering RNA (SIRNA) finds and attaches to the mRNA taking advantage of the natural pathway with natural cellular processes and gene specific inhibition of expression.

Kay Crabtree voiced the information in a clear and concise manner and presented the information in an understandable and comprehensible format.

Clinical Trials: presented by Melissa Fladseth

Methods of delivery in gene therapy are "Exvivo" or outside of the patient, "Gene Transfer" and "Viral Vectors." Before a clinical trial with human involvement the plans are reviewed by physicians as well as the Internal Ethics and Safety Review Board (IRB) and the Recombinant DNA Advisory Committee of NIH (RAC). The Food and Drug Administration (FDA) is also involved in the testing and approval. Phase One is the assessment.....

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