You are designing a somatic cell gene therapy protocol for spinal muscular atrophy, a genetic disorder that is caused by a defect in the Survival of Motor Neurons (SMN) gene. You have cloned the SMN cDNA.
(a) What is the minimal cis-acting DNA regulatory element you would need to ligate to the SMN cDNA to get transcription of the cDNA in cells?
(b) Assume that you decide to use retroviral-mediated gene transfer to introduce the SMN cDNA into the patient's target cells ex vivo. What are the advantages and disadvantages of this choice of vector for gene delivery? Is there another vector that would work better?

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The Answer of You are designing a somatic cell gene therapy protocol for spinal muscular atrophy, a genetic disorder that is caused by a defect in the Survival of Motor Neurons (SMN) gene. You have cloned the SMN cDNA.
(a) What is the minimal cis-acting DNA regulatory element you would need to ligate to the SMN cDNA to get transcription of the cDNA in cells?
(b) Assume that you decide to use retroviral-mediated gene transfer to introduce the SMN cDNA into the patient's target cells ex vivo. What are the advantages and disadvantages of this choice of vector for gene delivery? Is there another vector that would work better?

You Are Designing a Somatic Cell Gene Therapy Protocol for Spinal