Search Results - "gene therapies"

Editing the human genome with CRISPR/Cas: a review of its molecular basis, current clinical applications, and bioethical implications by Miguel Ahumada-Ayala, Regina Aguilar-López, Nicolai González-Stoylov, Esmeralda Palacio-Sosa, David E. Cervantes-Barragán, Liliana Fernández-Hernández

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A pipeline for identifying guide RNA sequences that promote RNA editing of nonsense mutations that cause inherited retinal diseases by Nina Schneider, Ricky Steinberg, Amit Ben-David, Johanna Valensi, Galit David-Kadoch, Zohar Rosenwasser, Eyal Banin, Erez Y. Levanon, Dror Sharon, Shay Ben-Aroya

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A MicroRNA124 Target Sequence Restores Astrocyte Specificity of gfaABC1D-Driven Transgene Expression in AAV-Mediated Gene Transfer by Grit Taschenberger, Julia Tereshchenko, Sebastian Kügler

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Development and characterization of a non-human primate model of disseminated synucleinopathy by Alberto J. Rico, Alberto J. Rico, Alberto J. Rico, Almudena Corcho, Julia Chocarro, Julia Chocarro, Julia Chocarro, Goiaz Ariznabarreta, Goiaz Ariznabarreta, Goiaz Ariznabarreta, Elvira Roda, Elvira Roda, Elvira Roda, Adriana Honrubia, Adriana Honrubia, Adriana Honrubia, Patricia Arnaiz, Patricia Arnaiz, Patricia Arnaiz, José L. Lanciego, José L. Lanciego, José L. Lanciego

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CRISPR Gene-Editing Models Geared Toward Therapy for Hereditary and Developmental Neurological Disorders by Poh Kuan Wong, Fook Choe Cheah, Saiful Effendi Syafruddin, M. Aiman Mohtar, Norazrina Azmi, Pei Yuen Ng, Eng Wee Chua

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Robust Long-term Transduction of Common Marmoset Neuromuscular Tissue With rAAV1 and rAAV9 by Hironori Okada, Hidetoshi Ishibashi, Hiromi Hayashita-Kinoh, Tomoko Chiyo, Yuko Nitahara-Kasahara, Yukihiro Baba, Sumiko Watanabe, Shin'ichi Takeda, Takashi Okada

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Managing Severe Hemophilia A in Children: Pharmacotherapeutic Options by Regling K, Callaghan MU, Sidonio R Jr

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Adeno-Associated Virus Serotype 8-Mediated Genetic Labeling of Cholangiocytes in the Neonatal Murine Liver by Sanghoon Lee, Ping Zhou, Senyo Whyte, Soona Shin

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Stable Cavitation-Mediated Delivery of miR-126 to Endothelial Cells by Stephanie He, Davindra Singh, Hossein Yusefi, Brandon Helfield

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Potential Applications of Mitochondrial Therapy with a Focus on Parkinson's Disease and Mitochondrial Transplantation by Pranay Wal, Ankita Wal, Himangi Vig, Danish Mahmood, Mohd Masih Uzzaman Khan

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Ultrasound Assisted Exosomal Delivery of Tissue Responsive mRNA for Enhanced Efficacy and Minimized Off-Target Effects by Wenqi Sun, Changyang Xing, Lianbi Zhao, Ping Zhao, Guodong Yang, Lijun Yuan

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Inhibition of Murine Cytomegalovirus Infection in Animals by RNase P-Associated External Guide Sequences by Wei Li, Jingxue Sheng, Mengqiong Xu, Gia-Phong Vu, Zhu Yang, Yujun Liu, Xu Sun, Phong Trang, Sangwei Lu, Fenyong Liu

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Combining Engineered U1 snRNA and Antisense Oligonucleotides to Improve the Treatment of a BBS1 Splice Site Mutation by Saskia Breuel, Mariann Vorm, Anja U. Bräuer, Marta Owczarek-Lipska, John Neidhardt

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Pharmacological intervention in hepatic stellate cell activation and hepatic fibrosis by Hans-Theo eSchon, Matthias eBartneck, Erawan eBorkham-Kamphorst, Jacob eNattermann, Twan eLammers, Frank eTacke, Ralf eWeiskirchen

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Cystic fibrosis treatment; small molecule modulators vs genetic therapy by Salma Aly Elmallah, Raafat Soliman

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CRISPR/Cas9-Mediated Deletion of CTG Expansions Recovers Normal Phenotype in Myogenic Cells Derived from Myotonic Dystrophy 1 Patients by Claudia Provenzano, Marisa Cappella, Rea Valaperta, Rosanna Cardani, Giovanni Meola, Fabio Martelli, Beatrice Cardinali, Germana Falcone

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Newborn Screening for Spinal Muscular Atrophy: Variations in Practice and Early Management of Infants with Spinal Muscular Atrophy in the United States by Craig M. Zaidman, Cameron D. Crockett, Ethan Wedge, Grace Tabatabai, Natalie Goedeker

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Multi-pathway Protective Effects of MicroRNAs on Human Chondrocytes in an In Vitro Model of Osteoarthritis by Rua Nader Al-Modawi, Jan E. Brinchmann, Tommy A. Karlsen

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FedExosomes: Engineering Therapeutic Biological Nanoparticles that Truly Deliver by Michelle E. Marcus, Joshua N. Leonard

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Role of Oxidative Stress in HIV-1-Associated Neurocognitive Disorder and Protection by Gene Delivery of Antioxidant Enzymes by Jean-Pierre Louboutin, David Strayer

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