Biochemistry 3385A Chapter Chapter 5: Gene Therapy
4 Feb 2015
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De ne terms used in gene therapy. Describe the sequenhal steps involved in gene therapy. Describe the sequenhal steps involved in producing complementary dna. Describe the sequenhal steps involved in dna synthesis by reverse transcriptase. Describe the retroviral life cycle. Explain problems with gene therapy. Never makes it to sperm or ooctyes. Types of delivery (in vivo) Lungs cf (ex vivo) Immune response can lead to death. De ni4ons: recombinant dna: genehcally engineered dna produced by combining dna from mulhple species. We don"t want it to be degraded: vector: vehicle for delivery into cells; viral and nonviral. Applies to diseases where three requirements are met: The speci c gene defect must be known. Defect must occur in single copy gene. A mutahon only happens in one gene such as gf. Target cells must be available and amenable to transfechon methods resulhng in long- term expression. Ex vivo gene therapy for ada- scid. First few trials used t- cells.
