Briefly explain two distinct cases of successfully commercialized gene therapy treatments, and two unsuccessful applications backed...

Briefly explain two distinct cases of successfully commercialized gene therapy treatments, and two unsuccessful applications backed up by reputable literature.

Expert Solution

Briefly explain two distinct cases of successfully commercialized gene therapy treatments, and two unsuccessful applications backed up by reputable literature.

Gene thrapy is the advanced metho and personalized treatment which involves interference of genes either to prevent or to treat the disease. It allows to treat the genetic disorder instead of using drugs or surgery.

The most successful gene therapies are to treat cancer and retinal disease. CAR T is Chimeric Antigen Receptor T Cell is the gene therapy to treat cancer marketed by Novartis' Kymriah. Spark’s Luxturna treates the error of RPE65 mutation-induced blindness enable to treat retinal disease. Both of the drugs are approved by FDA to enter into the market.

Glybera™ is the name of the gene therapy to treat lipoprotein lipase deficiency developed by uniQure, Inc.

GlaxoSmithKline (GSK) introduced gene therapy named as Strimvelis for SCID arises due to adenosine deaminase deficiency. Both the product faced similar difficult time whose applications are failed and unsuccessful.

Sydney Mullin answered 1 year ago

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