Which of the following conditions is most likely to be successfully treated by gene therapy using aviral vector to deliver a wild-type copy of one gene that is present in mutant form in a person withthe condition? Assume that the viral vector used has the ability to home to relevant target tissues.Also assume that the patient can be treated at a young enough age to avoid irreversible phenotypicimpacts of the mutation described.Timothy Syndrome, a multi-system disorder characterized by dysmorphic features and autistic behavioraltraits, caused by overexpression of calcium channel geneCerebral adrenoleukodystrophy (ALD), a neurological disorder caused by a frameshift mutation knocking outthe function of the ABCD1 gene encoding a lipid transporterOsteogenesis imperfecta caused by a dominant negative mutation in the collagen A1 geneMyopia (shortsightedness), a vision impairment with heritability estimates in the range of 0.6-0.8, where risk isimpacted by at least 200 genes

Answered: Which of the following conditions is…

Human Anatomy & Physiology (11th Edition)

11th Edition

ISBN:9780134580999

Author:Elaine N. Marieb, Katja N. Hoehn

Publisher:Elaine N. Marieb, Katja N. Hoehn

Chapter1: The Human Body: An Orientation

Section: Chapter Questions

Problem 1RQ: The correct sequence of levels forming the structural hierarchy is A. (a) organ, organ system,...

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Which of the following conditions is most likely to be successfully treated by gene therapy using a
viral vector to deliver a wild-type copy of one gene that is present in mutant form in a person with
the condition? Assume that the viral vector used has the ability to home to relevant target tissues.
Also assume that the patient can be treated at a young enough age to avoid irreversible phenotypic
impacts of the mutation described.
Timothy Syndrome, a multi-system disorder characterized by dysmorphic features and autistic behavioral
traits, caused by overexpression of calcium channel gene
Cerebral adrenoleukodystrophy (ALD), a neurological disorder caused by a frameshift mutation knocking out
the function of the ABCD1 gene encoding a lipid transporter
Osteogenesis imperfecta caused by a dominant negative mutation in the collagen A1 gene
Myopia (shortsightedness), a vision impairment with heritability estimates in the range of 0.6-0.8, where risk is
impacted by at least 200 genes

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Step 1: Introduction

"In investigating the potential applications of gene therapy, we consider different hereditary conditions and their suitability for treatment utilizing viral vectors to convey wild-type copies of genes. This examination aims to distinguish the condition most likely to advantage from gene treatment, taking into consideration the possibility of adjusting the basic hereditary defect and the potential for effective treatment, especially when administered at a young age."

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