Sentences with phrase «vivo gene therapy»

Strimvelis, which is the first approved ex vivo gene therapy worldwide, has been developed at SR - Tiget and brought to the market under a strategic alliance with GSK.

AveXis, Inc. and Genethon today announced they have entered into an exclusive, worldwide license agreement for in vivo gene therapy delivery of AAV9 vector into the central nervous system (CNS) for the treatment of spinal muscular atrophy (SMA).

Aiuti A, Roncarolo MG, Naldini L. Gene therapy for ADA - SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products.

In most previous in vivo gene therapy experiments, researchers have used viruses to carry the DNA into the patient's tissues.

New delivery technique used to create a glowing adult mouse potentially opens the door for in vivo gene therapy

Not exact matches

After preclinical studies, a gene therapy trial for SCID - X1 was initiated, based on the use of complementary DNA containing a defective γc Moloney retrovirus — derived vector and ex vivo infection of CD34 + cells.

Intellia is developing human gene and cell therapies for both ex vivo and in vivo applications using CRISPR - Cas9 gene editing technology.

An AAV gene therapy approach has shown in preclinical in vivo models to produce stable gene expression, enhanced survival, and prevention of hypoglycemia.

Fundamental Science Overview Gene and gene - modified cell therapies treat diseases by delivering therapeutic DNA into a patient's cells, either in (in vivo) or outside his or her body (ex viGene and gene - modified cell therapies treat diseases by delivering therapeutic DNA into a patient's cells, either in (in vivo) or outside his or her body (ex vigene - modified cell therapies treat diseases by delivering therapeutic DNA into a patient's cells, either in (in vivo) or outside his or her body (ex vivo).

GlaxoSmithKline has sold a number of gene therapy products — including its approved autologous ex vivo product Strimvelis — to Orchard Therapeutics.

Zonari E, Desantis G, Petrillo C, Boccalatte FE, Lidonnici MR, Kajaste - Rudnitski A, Aiuti A, Ferrari G, Naldini L, Gentner B. Efficient ex vivo engineering and expansion of highly purified human hematopoietic stem and progenitor cell populations for gene therapy.

In particular, Daniel Scherman has discovered techniques for the in vivo delivery of DNA plasmids for gene therapy and genetic vaccination.

The current competence fields and main contributions of Daniel Scherman concern drug gene therapy, drug delivery, in vivo imaging, and rare drug diseases.