In humans, the absence of an enzyme called purine nucleoside phosphorylase (PNP) results in a severe T-cell

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In humans, the absence of an enzyme called purine nucleoside phosphorylase (PNP) results in a severe T-cell immunodeficiency similar to that of severe combined immunodeficiency disease (SCID). PNP deficiency exhibits an autosomal recessive pattern of inheritance, and the gene encoding human PNP has been cloned and sequenced. Would PNP deficiency be a good candidate for treatment by gene therapy? Design a procedure for the treatment of PNP deficiency by somatic-cell gene therapy.

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Principles of Genetics

ISBN: 978-1119142287

7th edition

Authors: D. Peter Snustad, Michael J. Simmons

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