Which of the following statements aboutsomatic cell gene therapy (SCGT) are correct?
A. In somatic cell gene therapy (SCGT), the therapeutic genes are transferred into any cell other than a gamete, germ cell, gametocyte, or undifferentiated stem cell.
B. Any such modifications affect the individual patient only, and are not inherited by offspring.
C. Somatic gene therapy represents mainstream basic and clinical research, in which therapeutic DNA (either integrated in the genome or as an external episome or plasmid) is used to treat disease.
D. Most focus on severe genetic disorders, including immunodeficiencies, hemophilia, thalassemia, and cystic fibrosis. Such single gene disorders are good candidates for somatic cell therapy.
Which of the following statements about germline gene therapy (GGT) are correct?
A. GGT involves the introduction of new genetic material into either germ cells (sperm or oocytes) or into zygotes in vitro prior to fertilization or implantation.
B. In GGT, germ cells (sperm or egg cells) are modified by the introduction of functional genes into their genomes.
C. Modifying a germ cell causes all the organism's cells to contain the modified gene. The change is therefore heritable and passed on to later generations.
D. Australia, Canada, Germany, Israel, Switzerland, and the Netherlands prohibit GGT for application in human beings, for technical and ethical reasons, including insufficient knowledge about possible risks to future generations and higher risks versus somatic cell gene therapy (SCGT).
Which of the following statements about 'gene therapy by the US FDA are correct?
A. Gene therapy is products that mediate their effects by transcription and/or translation of transferred genetic material and/or by integrating into the host genome and that are administered as nucleic acids, viruses, or genetically engineered microorganisms.
B. The products may be used to modify cells in vivo or transferred to cells ex vivo prior to administration to the recipient.
C. In gene therapy, scientists can do one of several things depending on the problem that is present. They can replace a gene that causes a medical problem with one that doesn't, add genes to help the body to fight or treat disease, or turn off genes that are causing problems.
D. Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use.
Which of the following statements about the definition of 'gene therapy' byFarlex Partner Medical Dictionary are correct?
A. Gene therapy is alteration of somatic or germ-line DNA to correct or prevent disease; the process of inserting a gene artificially into the genome of organism to genetic defect or to add a new biologic property or function with therapeutic potential.
B. In somatic gene therapy, functional DNA sequences are inserted into cells that lack a specific gene or bear a faulty version of it. Vectors include replication-defective viruses, liposomes, and plasmids.
C. For transfer of genetic material by viral infection (called transduction), retroviruses are particularly suitable as vectors because their RNA, converted to DNA by reverse transcriptase, becomes part of the genome of the infected cell. Adenovirus and herpesvirus are also used.
D. Progress has been made in treating several inherited disorders, including severe combined immunodeficiency, cystic fibrosis, and hemophilia B.
Whichofthefollowingstatementsaboutthedefinitionof'genetherapy'byFarlexPartnerMedicalDictionaryarecorrect?
A. Gene therapy has several applications in oncology, including the transduction into malignant tumor cells of genes encoding cytokines or coactivation factors to augment host antitumor responses and the transfer of tumor suppressor genes, particularly p53 (the most commonly mutated gene found in human cancers), to enhance the sensitivity of malignant cells to chemotherapeutic agents.
B. Use of viral vectors is associated with a risk of localized and systemic inflammation mediated by cytokines, which can be fatal.
C. Germ-line therapy inserts specific genes directly into the DNA of sperm, egg, or embryo, producing heritable alterations of the genome.
D. Progresshasbeenmadeintreatingseveralinheriteddisorders,includingseverecombinedimmunodeficiency,cysticfibrosis,andhemophiliaB.
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