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Gene transfer and gene therapy

Published on March 18, 2015   58 min

A selection of talks on Haematology

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0:00
This is Dave Williams, I'm chief of hematology/oncology at Boston Children's Hospital and Dana Farber Cancer Institute. And I'm going to be talking today about gene transfer and gene therapy.
0:14
So what I would like to cover quickly today is outlined in the first slide. We'll talk a little bit about the definition of gene therapy, the requirements for success of gene therapy, in particular focusing on blood diseases as a model system, talk just briefly about applicable vector systems that can be used in blood diseases including gamma retroviruses, lentiviruses, foamy and alpha retroviruses, then briefly review experience from clinical trials, including the adenosine deaminase or ADA-SCID trial, the X-SCID trial, CGD, WAS, CCALD trial, and beta-thalassemia, and finally, talk about insertional mutagenesis as it relates to these clinical trials and experience with leukemias, and end with a couple slides on prospects and new approaches, including new cell targets and gene editing.
1:11
OK, so the first slide is just a general introduction to gene therapy by way of definition. So we think of gene therapy as introducing new genetic material into the cells of an organism for therapeutic purposes. There's essentially two broad concepts or types: germline, in which the DNA is introduced in the germ cells and therefore the new material can be passed into the gene pool, and somatic, in which the gene sequences are introduced into specialized somatic cells, and therefore the genetic material is really limited to the individual recipient. And of course, the former germline gene therapy is not practiced; in fact is banned in most countries, and the latter is what we'll be focusing on today.

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