Compositions and methods for treatment of type vii collagen deficiencies
A technology of collagen and lentiviral vectors, applied in biochemical equipment and methods, drug combinations, and the use of vectors to introduce foreign genetic materials, etc., can solve problems such as no priority integration
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example 1
[0134] A. Illustration of the structure and characterization of FCX-007 transduced with the lentiviral vector INXN-2002
[0135] FCX-007 cells derived from the lentiviral vector INXN-2002 cells were suspended in a cryopreservation medium consisting of Iscove's Modified Dulbecco's Medium (IMDM) without fetal bovine serum (50.0% ), Profreeze-CDMTM (42.5%) and dimethylsulfoxide (DMSO) (7.5%). The structural characterization of FCX-007 drug product (DS) includes the primary structure of autologous human dermal fibroblasts (HDF) and the structure of the lentiviral vector used to transduce and genetically modify HDF cells.
[0136] B. Lentiviral vector (INXN-2002)
[0137] The INXN-2002 lentiviral vector (LV) used to transduce and introduce human collagen 7A1 gene into HDF cells is a recombinant lentiviral vector encoding human collagen 7A1 gene. INXN-2002 LV is a self-inactivating (SIN) lentiviral vector constructed based on human immunodeficiency virus type 1 (HIV-1) pseudotyped...
example 2
[0188] Example 2: FCX-007 transduced with INXN-2004 lentiviral vector
[0189]FCX-007 is an autologous fibroblast product genetically modified by the INXN-2004 lentiviral vector (LV-COL7) to express human collagen 7 protein (C7). The materials described below for the production of FCX-007 DS / INXN-2004 were similar to those described above in Example 1, except that the IGE-230 LV-COL7 vector plasmid was used to produce INXN-2002. The IGE-308 LV-COL7 vector plasmid was used to prepare the INXN-2004 vector. The 293 WCB cell line was co-transfected with the same helper plasmids pCMV-G, pCMV-Rev2 and pCgp.
[0190] INXN-2004 lentiviral vector
[0191] IGE308 LV-COL7 vector transfer plasmid
[0192] The IGE308 plasmid was constructed using standard molecular cloning methods. The construction process included cloning the human COL7A1 gene and introducing the cloned COL7A1 gene into the lentiviral vector (SIN) backbone pFUGW to generate the IGE308 LV-COL7 vector transfer plasmid. ...
example 3
[0273] Example 3: Training runs 8, 9 and 10 and enhanced enzymatic digestion of biopsies
[0274] Training Run 8 and Enhanced Enzymatic Digestion of Biopsies
[0275] In the TR mentioned above, it was suspected that the biopsy digestion method could not achieve efficient digestion of biopsy tissue with a size of 3-4 mm. In TR8, the application of additional shear forces during digestion was incorporated into the process to increase overall digestion efficiency. The digestion procedure was modified as follows: every 15 ± 2 minutes during digestion, pulse vortex the centrifuge tube for 5 seconds at the maximum setting, returning the centrifuge tube to the orbital shaker after each vortex; end the incubation at 60 minutes , pulse vortex the centrifuge tube for 10 seconds at the maximum setting.
[0276]Biopsies from RDEB donors were processed and digested using the enhanced digestion method. Cells from the digestion were seeded into T-75 flasks using medium supplemented with G...
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