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Lentiviral vectors pseudotyped with mutant BaEV glycoproteins

A viral vector, pseudotyping technology, applied in the direction of virus/bacteriophage, introduction of foreign genetic material, virus using vector, etc., can solve problems such as restriction and ineffective entry of glycoproteins

Active Publication Date: 2014-10-01
INST NAT DE LA SANTE & DE LA RECHERCHE MEDICALE (INSERM) +1
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

In addition, the RD114 / TR glycoprotein does not efficiently enter murine cells
Therefore, this receptor specificity limits the potential of this LV to act, since most preclinical disease models suitable for gene therapy are mouse models

Method used

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  • Lentiviral vectors pseudotyped with mutant BaEV glycoproteins
  • Lentiviral vectors pseudotyped with mutant BaEV glycoproteins
  • Lentiviral vectors pseudotyped with mutant BaEV glycoproteins

Examples

Experimental program
Comparison scheme
Effect test

specific Embodiment approach

[0150] The following examples demonstrate the advantageous properties of BaEV / TR- and BaEVRLess pseudotyped lentiviral vector particles for use as gene transfer vectors compared to previous pseudotyped lentiviral vectors.

[0151] Materials and Methods

[0152] Generation and titration of lentiviral vectors

[0153] Self-inactivating HIV-1 -derived vectors were generated by transient transfection of HEK293T cells (American Type Culture Collection, Rockville, MD, CRL-1573). 24 hours before transfection, the 2.610 6 HEK293T cells were seeded to 10-cm 2 on tissue culture dishes. 293T cells were grown in Dulbecco's modified Eagle's medium (DMEM, Invitrogen) supplemented with 10% fetal calf serum (FCS). Using 8.6 μg Gag-Pol packaging construct 8.91 and HIV-1 derived SIN transfer vector pHIV-SFFV-GFP-SIN encoding GFP and 2.5 μg pMD.G encoding VSV-G glycoprotein (GP) or 7 μg phCMV-RD114 / Cells were transfected with TR, phCMV-BaEVWT, phCMV-BaEV / TR, or phCMV-BaEVRLess by calcium...

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Abstract

The present invention concerns a pseudotyped viral vector particle for transferring biological material into cells, wherein said vector particle comprises at least: - a chimeric envelope glycoprotein which comprises or consists in a fusion of the transmembrane and extracellular domain of a baboon endogenous retrovirus (BaEV) envelope glycoprotein and the cytoplasmic tail domain of a murine leukemia virus (MLV) envelope glycoprotein; or - a modified BaEV envelope glycoprotein wherein the cytoplasmic tail domain is devoid of the fusion inhibitory R peptide.

Description

technical field [0001] The present invention relates to a pseudotyped lentiviral vector capable of efficient gene transfer to hematopoietic cells. Background technique [0002] Gene therapy holds promise for the cure of many inherited and acquired diseases, such as its use in the treatment of X-linked severe combined immunodeficiency (SCID-X1), adenosine deaminase (ADA) deficiency, and chronic granulomatous disease proven by success. Recently, successful treatment of patients with X-linked adrenoleukodystrophy (ALD) using lentiviral vectors was reported. In this trial, hematopoietic stem cell (HSC)-based gene therapy was able to halt disease progression in two patients with this fatal demyelinating disease of the central nervous system. Importantly, to correct all these defects of the hematopoietic system, therapeutic genes must be delivered to cells that are both capable of self-renewal and differentiation into all hematopoietic lineages. Since HSCs meet these criteria, ...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C12N15/867C07K14/15A61K48/00
CPCC12N15/86C12N2740/15043A61K48/00C12N2740/15045C12N2740/16045C07K14/005C12N2810/6054C07K2319/00C12N2740/16043C12N2740/13022A61K48/005C12N5/16C12N15/867
Inventor 阿奈·吉拉尔-加涅潘埃尔莎·费尔赫芬迪米特里·拉维尔特弗朗西斯-卢瓦克·科塞
Owner INST NAT DE LA SANTE & DE LA RECHERCHE MEDICALE (INSERM)
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