Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice

Autor: Adam C. Wilkinson, Daniel P. Dever, Ron Baik, Joab Camarena, Ian Hsu, Carsten T. Charlesworth, Chika Morita, Hiromitsu Nakauchi, Matthew H. Porteus

Publikováno v: Nature Communications, Vol 12, Iss 1, Pp 1-9 (2021)

CRISPR mediated gene correction of sickle cell disease (SCD) in patient-derived hematopoietic stem cells is a promising avenue for therapy. Here the authors use a humanized SCD mouse model to study gene editing in the context of autologous transplant

Externí odkaz: https://doaj.org/article/33cf7e3f25534b81a1ff9bc124c467e5

Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting

Autor: Carsten T. Charlesworth, Joab Camarena, M. Kyle Cromer, Sriram Vaidyanathan, Rasmus O. Bak, Jason M. Carte, Jason Potter, Daniel P. Dever, Matthew H. Porteus

Publikováno v: Molecular Therapy: Nucleic Acids, Vol 12, Iss , Pp 89-104 (2018)

Engineered nuclease-mediated gene targeting through homologous recombination (HR) in hematopoietic stem and progenitor cells (HSPCs) has the potential to treat a variety of genetic hematologic and immunologic disorders. Here, we identify critical par

Externí odkaz: https://doaj.org/article/c3df848dbdcf49debfbcacf34af2f508

Outcomes and complications of anal sacculectomy for non‐neoplastic anal sac disease in cats: 8 cases (2006–2019)

Autor: D Anderson, T Charlesworth, J C Jimeno Sandoval

Publikováno v: Journal of Small Animal Practice. 63:56-61

Objectives To describe the features of non-neoplastic anal sac disease in cats, the surgical procedure, complications, surgical outcome and prognosis compared to well-established data for anal sacculectomy in canine patients. Materials and methods A

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9d44b039e42ed9f9ed99446824eb5fc1
https://doi.org/10.1111/jsap.13414

Immunological barriers to haematopoietic stem cell gene therapy

Autor: Carsten T. Charlesworth, Ian Hsu, Adam C. Wilkinson, Hiromitsu Nakauchi

Publikováno v: Nature reviews. Immunology. 22(12)

Cell and gene therapies using haematopoietic stem cells (HSCs) epitomize the transformative potential of regenerative medicine. Recent clinical successes for gene therapies involving autologous HSC transplantation (HSCT) demonstrate the potential of

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f085af18968ae1546c705303732b1c4f
https://pubmed.ncbi.nlm.nih.gov/35301483

Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease

Autor: Helen Segal, Sruthi Mantri, M. Kyle Cromer, Neehar Bhatia, Maria Grazia Roncarolo, Annalisa Lattanzi, Ciaran M. Lee, David DiGiusto, Rasmus O. Bak, Carsten T. Charlesworth, Josefin Kenrick, Jason Skowronski, Matthew H. Porteus, J. Fraser Wright, Richard L. Frock, Daniel P. Dever, Narae Talbott, Christopher A. Vakulskas, Joab Camarena, Gang Bao, Waracharee Srifa, Premanjali Lahiri, John F. Tisdale

Publikováno v: Sci Transl Med
Lattanzi, A, Camarena, J, Lahiri, P, Segal, H, Srifa, W, Vakulskas, C A, Frock, R L, Kenrick, J, Lee, C, Talbott, N, Skowronski, J, Cromer, M K, Charlesworth, C T, Bak, R O, Mantri, S, Bao, G, DiGiusto, D, Tisdale, J, Wright, J F, Bhatia, N, Roncarolo, M G, Dever, D P & Porteus, M H 2021, ' Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease ', Science Translational Medicine, vol. 13, no. 598, eabf2444 . https://doi.org/10.1126/scitranslmed.abf2444

Sickle cell disease (SCD) is the most common monogenic serious disease with 300,000 births annually worldwide. SCD is autosomal recessive from a single point mutation in codon six of the β-globin gene (HBB) resulting in sickle hemoglobin. Ex vivo β

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::664c1c185e0b70b2cf6b0e1b79df95cb
https://doi.org/10.1126/scitranslmed.abf2444