Exam 20: Genetic Testing and Treatment

Newborn screening reveals that newborn Jessica has inherited PKU.Her parents are distraught at the diagnosis,but a nutritionist explains that Jessica can be treated,right away.The treatment for PKU is

Diagnosis of hereditary hemochromatosis cannot be based on the results of a genetic test alone because

Introducing a gene in a liposome through holes bored into the skull is a(n)_______ gene therapy.

The species naturally affected by Leber's congenital amaurosis II that led to development of gene therapy is

Macy has BRCA1 breast cancer.Her sister Philene is healthy,but wants to know if she has inherited the BRCA1 mutation that could make her develop cancer.She is a candidate for a

A patient received bone marrow modified by an adeno-associated virus (AAV)carrying the human gene that encodes an enzyme her body could not make.This is an example of

Sheree is referred to a genetic counselor because a cystic fibrosis (CF)test done as a routine part of her prenatal care indicated that she is a carrier of the most common mutant allele.Sheree is stunned,because no one in her family has the disease.She is 26 years old.The genetic counselor would most likely

An effective vector to genetically alter respiratory cells might be

Which of the following is a vector used to deliver genes in human gene therapy?

Enzyme replacement therapy treats

Performing gene therapy on somatic cells removed from the body and then returned to the body is called _______ gene therapy.

Which of the following would provide the longest lasting treatment for adenosine deaminase (ADA)deficiency?

Tyrone has Gaucher disease.He receives recombinant glucocerebrosidase every other week in an infusion to prevent the symptoms.This treatment is an example of

Pharmacological chaperone therapy

Nonheritable gene therapy is performed on _______ cells.

A test offered on the Web by a direct-to-consumer genetic testing company genotypes a gene for ability to taste bitter substances.This test is not regulated by the Clinical Laboratory Improvement Amendments (CLIA)because

A serious limitation of using a retrovirus as a vector for gene therapy is that

Germline gene therapy would correct a genetic defect in

Genetic counseling to help patients with Huntington disease in their families decide if they wish to be tested generally

Gene therapy for Leber's congenital amaurosis II consists of