Lentiviruses, part of the Retroviridae family, are known for their slow replication and ability to integrate RNA into host DNA, leading to chronic infections. Notable lentiviruses include HIV and FIV, causing AIDS in humans and cats, respectively. The text delves into their use in gene therapy, highlighting clinical successes in treating immunodeficiencies and genetic disorders, and outlines the meticulous process of manufacturing lentiviral vectors for therapeutic applications.
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Lentiviruses belong to the Retroviridae family, known for their slow replication cycle and ability to integrate their RNA genome into host cells
Definition and Importance
Reverse transcription is the process of transcribing viral RNA into DNA, crucial for integration and a defining characteristic of all retroviruses
Role in Lentiviral Infection
Reverse transcription plays a critical role in the infection and persistence of lentiviruses by allowing them to integrate their genetic material into the host genome
The lentivirus genus is diverse, with species that infect a range of hosts and cause various diseases, including HIV, FIV, EIAV, and CAEV
A lentivirus particle is composed of an outer envelope, a protein capsid, two copies of single-stranded RNA, and essential enzymes such as reverse transcriptase, integrase, and protease
The envelope, derived from the host cell membrane, is studded with viral glycoproteins that mediate attachment and entry into target cells
Enzymes such as reverse transcriptase, integrase, and protease play critical roles in the lentiviral life cycle, including synthesizing viral DNA, inserting it into the host genome, and processing viral proteins
Lentiviruses have been repurposed as vectors in gene therapy, allowing for the stable integration of therapeutic genes into the host genome
Diseases Targeted by Lentiviral Gene Therapy
Lentiviral gene therapy has shown promise in treating a range of diseases, including immunodeficiencies, hematological disorders, and genetic conditions
Success Stories in Clinical Trials
Lentiviral gene therapy has shown success in clinical trials, with restored immune function in SCID-X1 patients, improved outcomes in patients with Beta-Thalassemia and Sickle Cell Disease, and restored auditory function in mice with genetic deafness
The production of lentiviral vectors for therapeutic use involves the engineering of safe, non-pathogenic vectors capable of carrying therapeutic genes, followed by the assembly, collection, and purification of recombinant lentiviral particles