Results 241 to 250 of about 89,643 (298)

Correction: Intravitreal injection of fibrillin 2 (Fbn2) recombinant protein for therapy of retinopathy in a retina-specific Fbn2 knock-down mouse model. [PDF]

Sci Rep
Zhang RX, Wen Y, Guo DD, Xu FR, Wang GM, Wang XR, Shi YW, Ding J, Jiang Q, Jiang WJ, Jonas JB, Bi HS.   +11 more
europepmc   +1 more source

Systemic, RPE-directed AAV-<i>tyrosinase</i> therapy restores ocular pigmentation in an OCA1 mouse model. [PDF]

Mol Ther Adv
Larimer-Picciani AM, Jacob LB, Sullinger KJ, Kriebel WG, Sahel JA, Byrne LC.   +5 more
europepmc   +1 more source

Combinatorial targeting of the miR-379/miR-410 cluster normalizes glucose and lipid homeostasis in male models of diabetes and obesity. [PDF]

Nat Commun
Gil-Lozano M, Simoes E, Wu M, Wiedemann T, Kwon Y, Sekar R, Morigny P, Troullinaki M, Merl-Pham J, Buettner M, Feuchtinger A, Jouffe C, Maida A, Hass D, Samaga D, Wolff G, Machado J, Giroud M, Klepac K, Cummins CL, Ríos García M, Weber P, Zeigerer A, Uhlenhaut NH, Szendroedi J, Blüher M, Herzig S.   +26 more
europepmc   +1 more source

Cell-type-specific repression reveals MC4R neurons as a site of action for ciliary obesity genes. [PDF]

Mol Metab
Diener Blake A, Zhang Z, Alexandre C, Gorayeb A, Jain M, Liu Q, Pattali R, Bednarska A, Kaur Pandher P, Zhang S, Yue X, Nuñez J, Vaisse C.   +12 more
europepmc   +1 more source

PERM1 Gene Therapy Prevents Pressure Overload-induced Heart Failure Through a Sarcomere-Mitochondria Energetic Microdomain

Warren J, Sreedevi K, Doku A, Zaitsev A, Montalvo R, Dennison C, Korte A, Salama S, Alabbi M, Tasneem S, Thomas R, Renton M, Edassery S, Deditz S, Burrows S, Patel G, Johnstone S, Yan Z, Smyth J, Kirk J.   +19 more
europepmc   +1 more source

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AAV as An Immunogen

Current Gene Therapy, 2007
The first in vivo adeno-associated viral vector (AAV) gene transfer experiments were performed in murine models of muscle directed gene transfer. These studies were remarkable for stable expression of a variety of immunogenic transgenes. These findings were translated to other target organs with multiple therapeutic gene products.
Luk H, Vandenberghe, James M, Wilson
openaire   +2 more sources