Zobrazeno 1 - 10
of 70
pro vyhledávání: '"Ciaran M. Lee"'
Autor:
Jessica L. Schneller, Ciaran M. Lee, Leah E. Venturoni, Randy J. Chandler, Ang Li, Sangho Myung, Thomas J. Cradick, Ayrea E. Hurley, William R. Lagor, Gang Bao, Charles P. Venditti
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 23, Iss , Pp 619-632 (2021)
Methylmalonic acidemia (MMA) is a metabolic disorder most commonly caused by mutations in the methylmalonyl-CoA mutase (MMUT) gene. Although adeno-associated viral (AAV) gene therapy has been effective at correcting the disease phenotype in MMA mouse
Externí odkaz:
https://doaj.org/article/40477017c9594449b2c47c26ce788c14
Autor:
Jerzy A. Woznicki, Nisha Saini, Peter Flood, Subhasree Rajaram, Ciaran M. Lee, Panagiota Stamou, Agnieszka Skowyra, Milan Bustamante-Garrido, Karine Regazzoni, Nyree Crawford, Simon S. McDade, Daniel B. Longley, Pedro Aza-Blanc, Fergus Shanahan, Syed A. Zulquernain, Jane McCarthy, Silvia Melgar, Bradford L. McRae, Ken Nally
Publikováno v:
Cell Death and Disease, Vol 12, Iss 10, Pp 1-15 (2021)
Abstract Rewiring of host cytokine networks is a key feature of inflammatory bowel diseases (IBD) such as Crohn’s disease (CD). Th1-type cytokines—IFN-γ and TNF-α—occupy critical nodes within these networks and both are associated with disrup
Externí odkaz:
https://doaj.org/article/3fd851ad37fa451687518ec09ff3247a
Autor:
Rajiv Sharma, Daniel P. Dever, Ciaran M. Lee, Armon Azizi, Yidan Pan, Joab Camarena, Thomas Köhnke, Gang Bao, Matthew H. Porteus, Ravindra Majeti
Publikováno v:
Nature Communications, Vol 12, Iss 1, Pp 1-12 (2021)
Genetic barcoding has been used to track clonal dynamics of cells. Here, the authors develop a Tracking Recombination Alleles in Clonal Engraftment using sequencing (TRACE-Seq), to barcode repaired alleles by introducing silent mutations or outside o
Externí odkaz:
https://doaj.org/article/24227f1bebe84818bfcc9151aa481b5d
Autor:
Ang Li, Ciaran M. Lee, Ayrea E. Hurley, Kelsey E. Jarrett, Marco De Giorgi, Weiqi Lu, Karol S. Balderrama, Alexandria M. Doerfler, Harshavardhan Deshmukh, Anirban Ray, Gang Bao, William R. Lagor
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 12, Iss , Pp 111-122 (2019)
Adeno-associated viral (AAV) vectors packaging the CRISPR-Cas9 system (AAV-CRISPR) can efficiently modify disease-relevant genes in somatic tissues with high efficiency. AAV vectors are a preferred delivery vehicle for tissue-directed gene therapy be
Externí odkaz:
https://doaj.org/article/312deba985e94bf79a24b835737e75c8
Autor:
Volker Wiebking, Ciaran M. Lee, Nathalie Mostrel, Premanjali Lahiri, Rasmus Bak, Gang Bao, Maria Grazia Roncarolo, Alice Bertaina, Matthew H. Porteus
Publikováno v:
Haematologica, Vol 106, Iss 3 (2020)
Allogeneic hematopoietic stem cell transplantation is an effective therapy for high-risk leukemias. In children, graft manipulation based on the selective removal of αβ T cells and B cells has been shown to reduce the risk of acute and chronic graf
Externí odkaz:
https://doaj.org/article/c38b71c2b182424abe1020cf6df23a42
Autor:
Haroon Butt, Ayman Eid, Zahir Ali, Mohamed A. M. Atia, Morad M. Mokhtar, Norhan Hassan, Ciaran M. Lee, Gang Bao, Magdy M. Mahfouz
Publikováno v:
Frontiers in Plant Science, Vol 8 (2017)
The CRISPR/Cas9 system has been applied in diverse eukaryotic organisms for targeted mutagenesis. However, targeted gene editing is inefficient and requires the simultaneous delivery of a DNA template for homology-directed repair (HDR). Here, we used
Externí odkaz:
https://doaj.org/article/a6a5bc086bbe476b94b1abb13e71d976
Autor:
Tanner, Rathbone, Ilayda, Ates, Lawrence, Fernando, Ethan, Addlestone, Ciaran M, Lee, Vincent P, Richards, Renee N, Cottle
Publikováno v:
The CRISPR Journal. 5:397-409
Adeno-associated virus vectors are the most used delivery method for liver-directed gene editing. Still, they are associated with significant disadvantages that can compromise the safety and efficacy of therapies. Here, we investigate the effects of
Autor:
Ciaran M. Lee, Charles P. Venditti, Jessica L. Schneller, Ang Li, Randy J. Chandler, Sangho Myung, Leah E. Venturoni, Gang Bao, Ayrea Hurley, Thomas J. Cradick, William R. Lagor
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 23, Iss, Pp 619-632 (2021)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
Methylmalonic acidemia (MMA) is a metabolic disorder most commonly caused by mutations in the methylmalonyl-CoA mutase (MMUT) gene. Although adeno-associated viral (AAV) gene therapy has been effective at correcting the disease phenotype in MMA mouse
Publikováno v:
Methods in molecular biology (Clifton, N.J.). 2429
Targeted genome editing in hematopoietic stem and progenitor cells (HSPCs) using CRISPR/Cas9 can potentially provide a permanent cure for hematologic diseases. However, the utility of CRISPR/Cas9 systems for therapeutic genome editing can be compromi
Publikováno v:
Nat Protoc
Genome editing using programmable nucleases is revolutionizing life science and medicine. Off-target editing by these nucleases remains a considerable concern, especially in therapeutic applications. Here we review tools developed for identifying pot