@article {2042,
	title = {Hardwiring tissue-specific AAV transduction in mice through engineered receptor expression.},
	journal = {Nat Methods},
	volume = {20},
	year = {2023},
	month = {2023 Jul},
	pages = {1070-1081},
	abstract = {<p>The development of transgenic mouse models that express genes of interest in specific cell types has transformed our understanding of basic biology and disease. However, generating these models is time- and resource-intensive. Here we describe a model system, SELective Expression and Controlled Transduction In Vivo (SELECTIV), that enables efficient and specific expression of transgenes by coupling adeno-associated virus (AAV) vectors with Cre-inducible overexpression of the multi-serotype AAV receptor, AAVR. We demonstrate that transgenic AAVR overexpression greatly increases the efficiency of transduction of many diverse cell types, including muscle stem cells, which are normally refractory to AAV transduction. Superior specificity is achieved by combining Cre-mediated AAVR overexpression with whole-body knockout of endogenous Aavr, which is demonstrated in heart cardiomyocytes, liver hepatocytes and cholinergic neurons. The enhanced efficacy and exquisite specificity of SELECTIV has broad utility in development of new mouse model systems and expands the use of AAV for gene delivery in vivo.</p>
},
	keywords = {Animals, Dependovirus, Gene Transfer Techniques, Genetic Therapy, Genetic Vectors, Mice, Mice, Transgenic, Transduction, Genetic, Transgenes},
	issn = {1548-7105},
	doi = {10.1038/s41592-023-01896-x},
	author = {Zengel, James and Wang, Yu Xin and Seo, Jai Woong and Ning, Ke and Hamilton, James N and Wu, Bo and Raie, Marina and Holbrook, Colin and Su, Shiqi and Clements, Derek R and Pillay, Sirika and Puschnik, Andreas S and Winslow, Monte M and Idoyaga, Juliana and Nagamine, Claude M and Sun, Yang and Mahajan, Vinit B and Ferrara, Katherine W and Blau, Helen M and Carette, Jan E}
}
