Lentiviral vectors are pivotal in gene therapy, offering the ability to correct genetic defects and create modified cells for cancer treatment. These vectors, derived from lentiviruses, are engineered to be safe, targeting both dividing and non-dividing cells. Their use in FDA-approved therapies like Kymriah and in treating genetic disorders showcases their medical significance.
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Lentiviral vectors are engineered viral vectors used in gene therapy that are derived from lentiviruses and are capable of delivering genetic material into both dividing and non-dividing cells
Ability to Deliver Genetic Material
Lentiviral vectors have the advantage of being able to deliver genetic material into both dividing and non-dividing cells, making them useful in gene therapy
Widely Used in Biomedical Research
Lentiviral vectors are widely used in biomedical research, including gene therapy and the creation of genetically modified cells for cancer therapy
Lentiviral vectors deliver genes by inserting the therapeutic gene into the vector's genome, which is then integrated into the host cell's genome by viral enzymes
The envelope protein of lentiviral vectors facilitates the vector's entry into specific cell types
Packaging proteins ensure the viral RNA genome is properly encapsulated in lentiviral vectors
Reverse transcriptase and integrase enzymes are critical components of lentiviral vectors, allowing for the delivery and integration of genetic material into host cells
Lentiviral vectors are designed with multiple safety features, such as self-inactivating vectors and a third-generation production system, to minimize potential risks
Lentiviral vectors are handled under Biosafety Level 2 conditions with trained personnel and appropriate safety equipment to prevent accidental exposure
The production of lentiviral vectors requires careful attention to maintain integrity, consistent transduction efficiency, and address biosafety concerns
Lentiviral vectors are used in the FDA-approved CAR T-cell therapy, Kymriah, to genetically modify T cells for cancer treatment
Lentiviral vectors have been used to restore immune function in patients with Wiskott-Aldrich syndrome and have potential for treating other genetic disorders
Lentiviral vectors are instrumental in creating transgenic animal models for studying human diseases and testing new treatments, showcasing their versatility and impact in advancing medical science
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In biomedical research, lentiviral vectors have been used to correct genetic defects, like in the treatment of ______ (ALD), and to engineer cells for cancer therapy.
adrenoleukodystrophy
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Initial step in lentiviral vector gene delivery
Insertion of therapeutic gene into vector genome.
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Role of reverse transcriptase in lentiviral vectors
Converts vector's RNA genome into DNA in target cells.
