MGY299Y1 Lecture Notes - Zinc Finger Nuclease, Zinc Finger, Transcription Activator-Like Effector Nuclease
3 Jun 2013
School
Department
Course
Professor
Document Summary
Currently most investigation with respect to gene therapy so for example, Gene therapy trials changed over the last decade. Use for genetic disease become more applied. Despite all promise of gene therapy, clogged up in phase trials most in phase 1, not looking at efficacy. Make double strand break, introduce wild-type sequence, repair by recombination theoretically. But side effects due to multiple repair mechanisms in some cells. Correcting mutation with respect to genetic disease get ribozymes to not only edit but change sequence exploit for therapy ex. , viruses can edit and change rna. Careful use giving artificial dose do not always know what arises from the dose. Other types of therapy slick and cool monoclonal type antibodies intrabodies derivatives of antibodies . Only work if there is amplification of this gene. If knows structure and thinks about biology of disease target various kinds of situations can block hiv infection by knowing the receptor.
