RP-L102 is Rocket's lentiviral
vector -based gene therapy in development for patients with FA with Rocket's collaboration partners at Centro de Investigaciones Energeticas, Medioambientales y Tecnologicas (CIEMAT) in Spain, CIBER-Rare Diseases and IIS-Fundacion Jimenez Diaz.
Oxford BioMedica will work with the UK Cystic Fibrosis Gene Therapy Consortium (GTC) and Imperial Innovations to develop a long-term therapy for patients with cystic fibrosis, focusing on a novel, replication deficient lentiviral
vector, in an inhaled formulation, to selectively introduce a CFTR gene into the relevant target cell.
The ability to switch on and off the expression of transgenes delivered via lentiviral
vectors is desirable in a number of experimental and therapeutic situations in which the transgene product must be regulated in a timely manner.
Methods: In this study, PRMT1 expression in the NSCLC cell line A549 was silenced using lentiviral
vector-mediated short hairpin RNAs.
In the present study, we constructed lentiviral
vectors to knock down NLRP3 to evaluate the role of the NLRP3 inflammasome in atherosclerosis in apolipoprotein (Apo) E-deficient mice, which can develop severe hypercholesterolemia and spontaneous atherosclerosis .
AMSBIO has announced a new range of ready-to-use lentiviral
particles that allow direct visualization of organelles or structures in cells without manipulation.
gene therapy technique, which was pioneered by Dr.
Led by Darrell Kotton, the scientists introduced gene-carrying lentiviral
vectors into the windpipe of mice.
Among their topics are targeted gene silencing in solid tumors with electrically mediated siRNA delivery, silencing virus replication, RNAi in the malaria vector Anopheles gambiae, lentiviral
and adeno-associated vector-based therapy for motor neuron disease through RNAi, studying autoimmunity by in-vitro RNA interference, and alternative splicing as a therapeutic target for human diseases.
United States Patent 6,555,342 B1 provides key intellectual property for the packaging of lentiviral
and retroviral gene transfer vectors.
The MPS-I program has shown clinical data in an ongoing proof-of-concept study in patients with the severe Hurler subtype, who were treated with ex vivo autologous HSC gene therapy, referred to as OTL-203, using a cryopreserved formulation and a lentiviral
(NASDAQ: RCKT) has inked a research agreement to support the clinical development of its lentiviral
vector -based gene therapy programmes in Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant Osteopetrosis towards registrational trials, the company said.