Sarepta总裁兼首席执行官Doug Ingram说:“Broad研究所发表的研究成果显示了MyoAAV具有作为突破性的新一代基因药物递送方法的潜力,并且这一结果已被Sarepta证实。与传统的AAV相比,MyoAAV高效率可使之在较低对数剂量有效地递送基因药物,这意味着MyoAAV可以大大降低未来的病毒载量和商品成本。” Sarepta执行副总裁、研发...
We suggest a de-novo protein design based approach to generating compact MYO7A replacement. Our approach generates a fully functional compact MYO7A enabling its packaging into a single AAV for potential therapeutic applications. Methods :Our approach is based on two key steps: modeling and ...
Altogether, these findings underline a complete amelioration of the XLCNM-like phenotypes in the Mtm1KO model at the structural and molecular levels upon DNM2 reduction through AAV-shRNA treatment and fully correlate with the rescue in muscle mass and force. Discussion Myotubular myopathy is the ...
AAV-mediated gene therapies can restore expression of essential proteins which are impaired in inherited disorders, leading to improvement in cardiac structure, function, and survival. Preclinical and Phase 1 clinical data have demonstrated robust safety and preliminary efficacy of RP-A501 (AAV9.LAMP2B...
The disclosure provides improved AAV -based, dual vector systems that facilitate the expression of full-length proteins whose coding sequences exceed that of the polynucleotide packaging capacity of an individual AAV vector. Described herein are modified hybrid dual vector systems that shift the coding...
An engineered AAV targeting integrin alpha V beta 6 presents improved myotropism across speciesGENE therapyADENO-associated virusSKELETAL muscleMUSCLE diseasesMETHODS engineeringCurrent adeno-associated virus (AAV) gene therapy using nature-derived AAVs is limited by non-optimal tissue targeting. In the ...
Subretinal delivery of AAV encoding human Myo7a rescues melanosome localization in sh1−/− RPE villi.Pasqualina, ColellaAndrea, SommellaElena, MarroccoUmberto, Di VicinoElena, PolishchukMarina, Garcia GarridoMathias, W. SeeligerRoman, Polishchuk...
We also utilized the Mtm1 KO mouse to determine the response to systemic intravenous delivery of an AAV serotype 9 vector expressing myotubularin under the muscle-specific desmin promoter. Myotubularin was rapidly and persistently expressed in muscles scattered throughout the body and this translated ...
-MOL THERChilders et al., " AAV-MTM1 Prolongs Survival and Rescues Severe Muscle Weakness in Mouse and Canine Models of X-Linked Myotubular Myopathy " 20(Supplement 1) Molecular Therapy (May 2012) (Childers I)
Myotubular myopathy (XLMTM) is a severe congenital disease that affects skeletal musculature, which is characterized by the presence of small myofibers with frequent occurrence of central nuclei. The disease is due to mutations in the MTM1 gene, encoding a phosphoinositide phosphatase named ...