The International Fanconi Anemia Gene Therapy Working Group helped the development of new generation of FA gene therapy programs, which began with a HIV-1-derived, self-inactivating lentiviral
Additionally, the collaboration may include an evaluation of Oxford BioMedica's Transgene Repression in vector Production (TRiP) System, and the development of stable producer cell lines for large-scale production of the lentiviral
To obtain lentiviral
particles, the 293 T packaging producer cells were cotransfected with the pLV/helper packaging plasmid mix (Invitrogen Life Technologies, USA) and the expression lentivector containing ExSi.P/PgkPuro, ExSi.P/Puro-EF1[alpha]-hTERT, TetIIP-EGFP-/Ubi-TetR, or TetIIP-GAL-EGFP-/Ubi-TetR plasmid, respectively, using Lipofectamine[TM] 2000 (Invitrogen Life Technologies, USA).
To determine the role of PRMT1 in NSCLC migration, we down-regulated the PRMT1 expression in the NSCLC cell line A549 via a lentiviral
vector-mediated small hairpin RNAs (shRNAs).
The positive clones were identified as lentiviral
vectors and pGCSIL/NLRP3-1 (sequence: 5'-7GGUGAAAUGUACUUAAAUC-3') induced the highest levels of downregulation.
Now using the new AMSBIO lentiviral
particles--researchers have an easy way to provide long term labels on many cellular structures (stable integration inside the genome) without the need for transfection reagents.
In a mouse model of emphysema, introduction into the windpipe of lentiviral
vectors carrying the gene responsible for making normal human alpha-1 antitrypsin led to sustained alpha-1 antitrypsin expression in the lung and reduced disease.
"Packaging RT and IN in trans, separately from the normal Gag-Pol polyprotein, adds a significant degree of safety to lentiviral
and retroviral vectors by minimizing the risk of generating a replication-competent virus," said John C.
The company multi-platform development approach applies the well-established lentiviral
vector and adeno-associated viral vector gene therapy platforms.
The new partnership brings together the academic partners leading expertise in developing gene therapy for CF and OXBs leading expertise in manufacturing lentiviral
vector-based therapies with Boehringer Ingelheims capabilities in drug discovery and the clinical development of novel breakthrough therapeutic agents.
Phase 2 clinical development plan for RP-L102, the company's lentiviral
vector, or LVV, -based gene therapy for the treatment of Fanconi Anemia, or FA.
ENPNewswire-August 28, 2019--Mustang Bio Announces License Agreement with CSL Behring for the Cytegrity Stable Producer Cell Line for the Production of MB-107 Lentiviral