Answered: 1) Since the process has not yet been…

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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Being able to understand the relationship between DNA, mRNA, tRNA, ribosomes and proteins, is necessary for any researcher interested in gene therapy. Gene therapy is dedicated to correcting genetic defects by replacing mutated genes with wildtype versions. This can include complete removal and introduction of a working copy of the entire gene (using knock-outs and knock-ins) or removal of only the actual mutated nucleotide. Sickle Cell Disease is an example of a recessive disorder based on a single-point mutation: GAG becomes GTG, which encodes a new amino acid. One would only need to replace a single base to correct the disorder making it very attractive to gene therapy researchers. The mutant sickle cell allele is called HbS (for sickled hemoglobin) while the wt allele is HbA. Pretend that Arcel Kasongo, a masters student from Cambridge, is studying for an oral exam on gene therapy, and has an especial interest in the disorder since he comes from the Democratic Republic of Congo where Sickle Cell has a historical high rate of occurrence due to the prevalence of malaria. Oral exams are a feature of certain British schools where students form a line and have to answer a series of questions from their proctor rather than take a written exam. Should we try oral exams at Lehman this semester?

1) Since the process has not yet been perfected, at what stage in the life of a Sickle Cell patient should gene therapy be attempted?

In the embryonic state only in order for all subsequent cells to inherit the wt allele.

In the fetal state in order for all subsequent cells to inherit the wt allele.

Since red blood cells are produced continuously by hemopoietic stem cells, the therapy could take place at any time in an afflicted person’s life, theoretically.

As soon as possible after the baby is born.

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