A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice
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Yang Yang | Deirdre McMenamin | Peter Bell | Kiran Musunuru | Hiroki Morizono | H. Morizono | Lili Wang | K. Musunuru | James M. Wilson | Hongwei Yu | M. Batshaw | P. Bell | Mark L. Batshaw | Hongwei Yu | Lili Wang | Zhenning He | John White | Chenyu Xu | Zhenning He | Deirdre McMenamin | John White | Chenyu Xu | Yang Yang | J. White | Peter Bell
[1] Morgan L. Maeder,et al. Characterization of Staphylococcus aureus Cas9: a smaller Cas9 for all-in-one adeno-associated virus delivery and paired nickase applications , 2015, Genome Biology.
[2] Y. Doyon,et al. In vivo genome editing of the albumin locus as a platform for protein replacement therapy. , 2015, Blood.
[3] James M. Wilson,et al. Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates. , 2015, Molecular therapy : the journal of the American Society of Gene Therapy.
[4] Martin J. Aryee,et al. Engineered CRISPR-Cas9 nucleases with altered PAM specificities , 2015, Nature.
[5] Zhiping Weng,et al. Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses. , 2015, Human gene therapy.
[6] S. Beer-Hammer,et al. In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency , 2015, Nature Biotechnology.
[7] David A. Scott,et al. In vivo genome editing using Staphylococcus aureus Cas9 , 2015, Nature.
[8] Florian Schmidt,et al. CRISPR genome engineering and viral gene delivery: A case of mutual attraction , 2015, Biotechnology journal.
[9] A. Parker,et al. Evidence for Contribution of CD4+CD25+ Regulatory T Cells in Maintaining Immune Tolerance to Human Factor IX following Perinatal Adenovirus Vector Delivery , 2015, Journal of immunology research.
[10] Martin J. Aryee,et al. GUIDE-Seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases , 2014, Nature Biotechnology.
[11] Yong Huang,et al. Promoterless gene targeting without nucleases ameliorates haemophilia B in mice , 2014, Nature.
[12] Feng Zhang,et al. Genome engineering using CRISPR-Cas9 system. , 2015, Methods in molecular biology.
[13] J. Doudna,et al. The new frontier of genome engineering with CRISPR-Cas9 , 2014, Science.
[14] Gang Wang,et al. Targeted and genome-wide sequencing reveal single nucleotide variations impacting specificity of Cas9 in human stem cells , 2014, Nature Communications.
[15] Pumin Zhang,et al. Efficient gene editing in adult mouse livers via adenoviral delivery of CRISPR/Cas9 , 2014, FEBS letters.
[16] M. Tuchman,et al. A longitudinal study of urea cycle disorders. , 2014, Molecular genetics and metabolism.
[17] E. Lander,et al. Development and Applications of CRISPR-Cas9 for Genome Engineering , 2014, Cell.
[18] Hao Yin,et al. Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype , 2014, Nature Biotechnology.
[19] Y. Doyon,et al. Robust ZFN-mediated genome editing in adult hemophilic mice. , 2013, Blood.
[20] R. Falahati,et al. Role of antigen-specific regulatory CD4+CD25+ T cells in tolerance induction after neonatal IP administration of AAV-hF.IX , 2013, Gene Therapy.
[21] M. Tuchman,et al. Clinical outcomes of neonatal onset proximal versus distal urea cycle disorders do not differ. , 2013, The Journal of pediatrics.
[22] Lili Wang,et al. Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector. , 2012, Human gene therapy.
[23] H. Morizono,et al. Sustained Correction of OTC Deficiency in Spf ash mice Using Optimized Self-complementary AAV2/8 Vectors , 2011, Gene Therapy.
[24] Lili Wang,et al. AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta). , 2011, Molecular therapy : the journal of the American Society of Gene Therapy.
[25] M. R. Delgado Alvira,et al. Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale. , 2010, Human gene therapy.
[26] R. Herzog,et al. Hepatic gene transfer as a means of tolerance induction to transgene products. , 2009, Current gene therapy.
[27] I. Alexander,et al. Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors. , 2008, Molecular therapy : the journal of the American Society of Gene Therapy.
[28] H. Morizono,et al. Long-term correction of ammonia metabolism and prolonged survival in ornithine transcarbamylase-deficient mice following liver-directed treatment with adeno-associated viral vectors. , 2006, Molecular therapy : the journal of the American Society of Gene Therapy.
[29] R. Jessberger,et al. Precise hit: adeno-associated virus in gene targeting , 2005, Nature Reviews Microbiology.
[30] T. Daly. AAV-mediated gene transfer to the liver. , 2004, Methods in molecular biology.
[31] Lili Wang,et al. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy , 2002, Proceedings of the National Academy of Sciences of the United States of America.
[32] H. Morizono,et al. Expression of Wild-Type and Mutant Human Ornithine Transcarbamylase Genes in Chinese Hamster Ovary Cells and Lack of Dominant Negative Effect of R141Q and R40H Mutants , 2000, Pediatric Research.
[33] Expression, purification and kinetic characterization of wild-type human ornithine transcarbamylase and a recurrent mutant that produces 'late onset' hyperammonaemia. , 1997, The Biochemical journal.
[34] A. Moorman,et al. Development of the ornithine cycle in rat liver: Zonation of a metabolic pathway , 1996, Hepatology.
[35] L. Rosenberg,et al. The spfash mouse: a missense mutation in the ornithine transcarbamylase gene also causes aberrant mRNA splicing. , 1989, Proceedings of the National Academy of Sciences of the United States of America.