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Lentivirus (lente-, Latin for "slow") is a genus of retroviruses, causing chronic and deadly diseases by long incubation periods in human or other mammalian species [1]. The virion is a medium-sized (80-100nm) and enveloped, slightly pleomorphic, spherical with an isometric nucleocapsid containing two copies of positive-sense ssRNA genome. Most lentiviral vectors are based on the Human Immunodeficiency Virus (HIV), which causes AIDS. To date, 5 serogroups have been recognized according to the vertebrate hosts they are associated with (primates, sheep and goats, horses, domestic cats, and cattle). Among them, the primate lentiviruses are distinguished by the utilization of CD4 surface protein as a receptor and the absence of dUTPase [2]. Considering the key safety concerns during the use of HIV-derived lentivirus vectors, recombinant lentivirus has been designed and widely used for gene delivery in most cell types.

Derived from HIV-1, lentiviruses can integrate a significant amount of viral cDNA into the host genome, mediate stable and long-term transgene expression, and efficiently infect dividing cells and nondividing cells, which makes lentivirus an attractive gene delivery vehicles in most cell types [3].

Table1. Selected clinical trials using lentivirus vectors.
Indication Transplants Delivery route Phase Sponsor
β-Thalassemia Autologous CD34+ cells transduced with Lenti-TNS9.3.55 Intravenous I Memorial Sloan Kettering Cancer Center
Autologous CD34+ cells transduced with LentiGlobin BB305 Intravenous bluebird bio
Autologous CD34+ cells transduced with LentiGlobin BB305 Intravenous I–II bluebird bio
Autologous CD34+ cells transduced with Lenti- β-globin Intravenous I–II IRCCS San Raffaele
X-linked adrenoleukodystrophy Lenti-ABCD1 Intracerebral I–II Shenzhen Geno-Immune Medical Institute
Autologous CD34+ cells transduced with Lenti-ABCD1 Intravenous I–II Shenzhen Second People's Hospital
Wiskott-Aldrich Syndrome Autologous CD34+ cells transduced with Lenti-WAS gene Intravenous I–II Genethon
Autologous CD34+ cells transduced with Lenti-WAS gene Intravenous II GlaxoSmithKline
Autologous CD34+ cells transduced with Lenti-WAS gene Intravenous I–II Genethon
Autologous CD34+ cells transduced with Lenti-WAS gene Intravenous I–II Genethon
Metachromatic leukodystrophy Lenti-ARSA intracerebral Not applicable Not applicable
Autologous CD34+ stem cells transduced with Lenti-ARSA Intravenous II GlaxoSmithKline
Autologous CD34+ stem cells transduced with Lenti-ARSA Intravenous I–II Shenzhen Second People's Hospital
Sickle cell disease Lenti-gamma-globin Intravenous I–II Children's Hospital Medical Center, Cincinnati
Autologous CD34+ stem cells transduced with Lenti-βAS3-FB Intravenous I Donald B. Kohn, M.D.
Autologous CD34+ cells transduced with LentiGlobin BB305 Intravenous I Bluebird bio
Autologous CD34+ cells transduced with Lenti-shBCL11a Intravenous I David Williams
Fanconi anemia Autologous CD34+ cells transduced with Lenti- FANCA Intravenous I Fred Hutchinson Cancer Research Center


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