Zobrazeno 1 - 10
of 23
pro vyhledávání: '"Annalisa Lattanzi"'
Autor:
Liwen Xu, Premanjali Lahiri, Jason Skowronski, Neehar Bhatia, Annalisa Lattanzi, Matthew H. Porteus
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 317-331 (2023)
Ex vivo gene correction with CRISPR-Cas9 and a recombinant adeno-associated virus serotype 6 (rAAV6) in autologous hematopoietic stem/progenitor cells (HSPCs) to treat sickle cell disease (SCD) has now entered early-phase clinical investigation. To f
Externí odkaz:
https://doaj.org/article/cc28c225e72749008b29603e06adf7f8
Autor:
Honglin Chen, Steffen Durinck, Hetal Patel, Oded Foreman, Kathryn Mesh, Jeffrey Eastham, Roger Caothien, Robert J. Newman, Merone Roose-Girma, Spyros Darmanis, Soren Warming, Annalisa Lattanzi, Yuxin Liang, Benjamin Haley
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 27, Iss , Pp 431-449 (2022)
With the aim of expediting drug target discovery and validation for respiratory diseases, we developed an optimized method for in situ somatic gene disruption in murine lung epithelial cells via AAV6-mediated CRISPR-Cas9 delivery. Efficient gene edit
Externí odkaz:
https://doaj.org/article/113e78dbbb674feba29efc5e54161173
Autor:
Sara Fañanas-Baquero, Oscar Quintana-Bustamante, Daniel P. Dever, Omaira Alberquilla, Rebeca Sanchez-Dominguez, Joab Camarena, Isabel Ojeda-Perez, Mercedes Dessy-Rodriguez, Rolf Turk, Mollie S. Schubert, Annalisa Lattanzi, Liwen Xu, Jose L. Lopez-Lorenzo, Paola Bianchi, Juan A. Bueren, Mark A. Behlke, Matthew Porteus, Jose-Carlos Segovia
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 22, Iss , Pp 237-248 (2021)
Pyruvate kinase deficiency (PKD), an autosomal-recessive disorder, is the main cause of chronic non-spherocytic hemolytic anemia. PKD is caused by mutations in the pyruvate kinase, liver and red blood cell (PKLR) gene, which encodes for the erythroid
Externí odkaz:
https://doaj.org/article/36126901876d48f9918fa4a741965bd8
Autor:
Annalisa Lattanzi, Stephanie Duguez, Arianna Moiani, Araksya Izmiryan, Elena Barbon, Samia Martin, Kamel Mamchaoui, Vincent Mouly, Francesco Bernardi, Fulvio Mavilio, Matteo Bovolenta
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 7, Iss C, Pp 11-19 (2017)
Exonic duplications account for 10%–15% of all mutations in Duchenne muscular dystrophy (DMD), a severe hereditary neuromuscular disorder. We report a CRISPR (clustered regularly interspaced short palindromic repeat)/Cas9-based strategy to correct
Externí odkaz:
https://doaj.org/article/88c9ce137af0434d9ad8ef36a9936d97
Autor:
Vasco Meneghini, Annalisa Lattanzi, Luigi Tiradani, Gabriele Bravo, Francesco Morena, Francesca Sanvito, Andrea Calabria, John Bringas, Jeanne M Fisher‐Perkins, Jason P Dufour, Kate C Baker, Claudio Doglioni, Eugenio Montini, Bruce A Bunnell, Krystof Bankiewicz, Sabata Martino, Luigi Naldini, Angela Gritti
Publikováno v:
EMBO Molecular Medicine, Vol 8, Iss 5, Pp 489-510 (2016)
Abstract Metachromatic leukodystrophy (MLD) and globoid cell leukodystrophy (GLD or Krabbe disease) are severe neurodegenerative lysosomal storage diseases (LSD) caused by arylsulfatase A (ARSA) and galactosylceramidase (GALC) deficiency, respectivel
Externí odkaz:
https://doaj.org/article/df4a76df4d1e46e5afca6e405d504c4a
Autor:
Nicole Armbruster, Annalisa Lattanzi, Matthieu Jeavons, Laetitia Van Wittenberghe, Bernard Gjata, Thibaut Marais, Samia Martin, Alban Vignaud, Thomas Voit, Fulvio Mavilio, Martine Barkats, Ana Buj-Bello
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Spinal muscular atrophy (SMA) is an autosomal recessive disease of variable severity caused by mutations in the SMN1 gene. Deficiency of the ubiquitous SMN function results in spinal cord α-motor neuron degeneration and proximal muscle weakness. Gen
Externí odkaz:
https://doaj.org/article/c3375dc222574ce59427a986034859f1
Autor:
Annalisa Lattanzi, Bernhard Gentner, Daniela Corno, Tiziano Di Tomaso, Pieter Mestdagh, Frank Speleman, Luigi Naldini, Angela Gritti
Publikováno v:
PLoS ONE, Vol 8, Iss 6, p e67411 (2013)
Several microRNAs (miRNAs) that are either specifically enriched or highly expressed in neurons and glia have been described, but the identification of miRNAs modulating neural stem cell (NSC) biology remains elusive. In this study, we exploited high
Externí odkaz:
https://doaj.org/article/9e3373cf83b042cfae582851bdaec61f
Autor:
Cécile Masson, Mario Amendola, Annarita Miccio, Annalisa Lattanzi, Ciaran M. Lee, Chiara Antoniani, Sophie Ramadier, Gang Bao, Olivier Alibeu, Vasco Meneghini, Tristan Felix, Matthew H. Porteus, Giulia Pavani, Fulvio Mavilio, Fatima Amor
Publikováno v:
Molecular Therapy
Molecular Therapy, Cell Press, 2019, 27, pp.137-150. ⟨10.1016/j.ymthe.2018.10.008⟩
Molecular Therapy, 2019, 27, pp.137-150. ⟨10.1016/j.ymthe.2018.10.008⟩
Molecular Therapy, Cell Press, 2019, 27, pp.137-150. ⟨10.1016/j.ymthe.2018.10.008⟩
Molecular Therapy, 2019, 27, pp.137-150. ⟨10.1016/j.ymthe.2018.10.008⟩
International audience; Editing the beta-globin locus in hematopoietic stem cells is an alternative therapeutic approach for gene therapy of beta-thalassemia and sickle cell disease. Using the CRISPR/Cas9 system, we genetically modified human hematop
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5877ed9da54aab838a3dc0587cdf6277
https://doi.org/10.1016/j.ymthe.2018.10.008
https://doi.org/10.1016/j.ymthe.2018.10.008
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
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
https://doi.org/10.1126/scitranslmed.abf2444
Autor:
Mollie S. Schubert, Mercedes Dessy-Rodriguez, Daniel P. Dever, Joab Camarena, Jose L. Lopez-Lorenzo, Rebeca Sanchez-Dominguez, Matthew H. Porteus, Rolf Turk, Annalisa Lattanzi, Isabel Ojeda-Perez, Oscar Quintana-Bustamante, Jose-Carlos Segovia, Liwen Xu, Mark A. Behlke, Paola Bianchi, Sara Fañanas-Baquero, Juan A. Bueren, Omaira Alberquilla
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 22, Iss, Pp 237-248 (2021)
Molecular Therapy: Methods & Clinical Development, Vol 22, Iss, Pp 237-248 (2021)
Pyruvate kinase deficiency (PKD), an autosomal-recessive disorder, is the main cause of chronic non-spherocytic hemolytic anemia. PKD is caused by mutations in the pyruvate kinase, liver and red blood cell (PKLR) gene, which encodes for the erythroid
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::79b9542cc523ced03183890ba4bdf24a
https://pubmed.ncbi.nlm.nih.gov/34485608
https://pubmed.ncbi.nlm.nih.gov/34485608