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Sort the items below into the gene therapy vector categories provided AAV vector lentiviral vector Therapeutic genes delivered by this vector are maintained extrachromosomally on an episome Used for ex vivo therapy Can deliver therapeutic genes up to 5kb Used for in vivo therapy Not good at homing to target tissues upon injection into a patient

Sort the items below into the gene therapy vector categories provided AAV vector lentiviral vector Therapeutic genes delivered by this vector are maintained extrachromosomally on an episome Used for ex vivo therapy Can deliver therapeutic genes up to 5kb Used for in vivo therapy Not good at homing to target tissues upon injection into a patient

Human Anatomy & Physiology (11th Edition)
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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Sort the items below into the gene therapy vector categories provided AAV vector lentiviral vector Therapeutic genes delivered by this vector are maintained extrachromosomally on an episome Used for ex vivo therapy Can deliver therapeutic genes up to 5kb Used for in vivo therapy Not good at homing to target tissues upon injection into a patient Mutations may occur when the therapeutic gene delivered by this vector integrates into the host cell chromosome Can deliver therapeutic genes up to 10kb Can home efficiently to various target tissues after injection into a patient
Transcribed Image Text:Sort the items below into the gene therapy vector categories provided AAV vector lentiviral vector Therapeutic genes delivered by this vector are maintained extrachromosomally on an episome Used for ex vivo therapy Can deliver therapeutic genes up to 5kb Used for in vivo therapy Not good at homing to target tissues upon injection into a patient Mutations may occur when the therapeutic gene delivered by this vector integrates into the host cell chromosome Can deliver therapeutic genes up to 10kb Can home efficiently to various target tissues after injection into a patient
Sort the items below according to whether they are relevant or necessary for two possible CRISPR-based gene therapy strategies for treatment of sickle cell anemia: (1) the strategy discussed in Video 5.2 targeting the BCL11A gene, a transcription factor that negatively regulates the expression of gamma globin in adults; and (2) an alternative strategy attempting to directly repair the missense mutation in the beta globin gene causing sickle cell anemia. strategy 1 (targeting BCL11A) strategy 2 (targeting the beta globin gene) non-homologous end joining a repair template containing wild type BCL11A sequence a repair template containing wild type beta globin sequence a guide RNA targeting an enhancer a guide RNA targeting the gamma globin gene a guide RNA targeting the E6V missense mutation aiming to maintain gamma globin gene expression in adults homology-directed repair
Transcribed Image Text:Sort the items below according to whether they are relevant or necessary for two possible CRISPR-based gene therapy strategies for treatment of sickle cell anemia: (1) the strategy discussed in Video 5.2 targeting the BCL11A gene, a transcription factor that negatively regulates the expression of gamma globin in adults; and (2) an alternative strategy attempting to directly repair the missense mutation in the beta globin gene causing sickle cell anemia. strategy 1 (targeting BCL11A) strategy 2 (targeting the beta globin gene) non-homologous end joining a repair template containing wild type BCL11A sequence a repair template containing wild type beta globin sequence a guide RNA targeting an enhancer a guide RNA targeting the gamma globin gene a guide RNA targeting the E6V missense mutation aiming to maintain gamma globin gene expression in adults homology-directed repair
Expert Solution
Step 1: Introduction.

Q 14: answer :-  Gene therapy is a promising field that holds the potential to treat a wide range of genetic and acquired diseases by introducing therapeutic genes into a patient's cells. Various gene therapy vectors are employed to deliver these genes effectively. Two prominent types of gene therapy vectors are adeno-associated virus (AAV) vectors and lentiviral vectors.








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