Sentences with phrase «vivo gene and cell 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.

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).

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.