Search Results - "Dilworth, Kimberley L."

Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing by Nguyen Tran, Minh Thuan, Mohd Khalid, Mohd Khairul Nizam, Wang, Qi, Walker, Jacqueline K. R., Lidgerwood, Grace E., Dilworth, Kimberley L., Lisowski, Leszek, Pébay, Alice, Hewitt, Alex W.

“…Precision genome engineering has dramatically advanced with the development of CRISPR/Cas base editing systems that include cytosine base editors and adenine…”
Get full text

Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2 by Cabanes-Creus, Marti, Westhaus, Adrian, Navarro, Renina Gale, Baltazar, Grober, Zhu, Erhua, Amaya, Anais K., Liao, Sophia H.Y., Scott, Suzanne, Sallard, Erwan, Dilworth, Kimberley L., Rybicki, Arkadiusz, Drouyer, Matthieu, Hallwirth, Claus V., Bennett, Antonette, Santilli, Giorgia, Thrasher, Adrian J., Agbandje-McKenna, Mavis, Alexander, Ian E., Lisowski, Leszek

“…Use of the prototypical adeno-associated virus type 2 (AAV2) capsid delivered unexpectedly modest efficacy in an early liver-targeted gene therapy trial for…”
Get full text

Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality by Cabanes-Creus, Marti, Ginn, Samantha L, Amaya, Anais K, Liao, Sophia H Y, Westhaus, Adrian, Hallwirth, Claus V, Wilmott, Patrick, Ward, Jason, Dilworth, Kimberley L, Santilli, Giorgia, Rybicki, Arkadiusz, Nakai, Hiroyuki, Thrasher, Adrian J, Filip, Adrian C, Alexander, Ian E, Lisowski, Leszek

“…Adeno-associated virus (AAV) vectors have become one of the most widely used gene transfer tools in human gene therapy. Considerable effort is currently being…”
Get full text

Systemic AAV8-mediated delivery of a functional copy of muscle glycogen phosphorylase (Pygm) ameliorates disease in a murine model of McArdle disease by McNamara, Elyshia L, Taylor, Rhonda L, Clayton, Joshua S, Goullee, Hayley, Dilworth, Kimberley L, Pinós, Tomàs, Brull, Astrid, Alexander, Ian E, Lisowski, Leszek, Ravenscroft, Gianina, Laing, Nigel G, Nowak, Kristen J

“…Abstract McArdle disease is a disorder of carbohydrate metabolism that causes painful skeletal muscle cramps and skeletal muscle damage leading to transient…”
Get full text

Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors by Westhaus, Adrian, Cabanes-Creus, Marti, Dilworth, Kimberley L, Zhu, Erhua, Salas Gómez, David, Navarro, Renina G, Amaya, Anais K, Scott, Suzanne, Kwiatek, Magdalena, McCorkindale, Alexandra L, Hayman, Tara E, Frahm, Silke, Perocheau, Dany P, Tran, Bang Manh, Vincan, Elizabeth, Wong, Sharon L, Waters, Shafagh A, Riddiough, Georgina E, Perini, Marcos V, Wilson, Laurence O W, Baruteau, Julien, Diecke, Sebastian, González-Aseguinolaza, Gloria, Santilli, Giorgia, Thrasher, Adrian J, Alexander, Ian E, Lisowski, Leszek

“…The liver is a prime target for gene therapies using recombinant adeno-associated viral vectors. Multiple clinical trials have been undertaken for this target…”
Get more information