You have studied about the "Bubble Baby", a condition that is practiced to keep the infection prone baby safe. What is the reason of being infected prone? Write about the available treatments for the disease and the overall details about gene therapy.

You have studied about the "Bubble Baby", a condition that is practiced to keep the infection prone baby safe. What is the reason of being infected prone? Write about the available treatments for the disease and the overall details about gene therapy.

Human Heredity: Principles and Issues (MindTap Course List)

11th Edition

ISBN:9781305251052

Author:Michael Cummings

Publisher:Michael Cummings

Chapter11: Genome Alterations: Mutation And Epigenetics

Section: Chapter Questions

Problem 6QP: Although it is well known that X-rays cause mutations, they are routinely used to diagnose medical...

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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…
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…
The first attempts at gene therapy began in 1990 with the treatment of a young girl with a genetic disorder abbreviated SCID. What does SCID stand for? In the context of SCID, what does ADA stand for?
Discuss the advantages and disadvantages of gene therapy. A limitedamount of funding is available for gene therapy research. Makea priority list of the three top diseases for which you would fundresearch. Discuss your choices.