Výsledky vyhledávání - "Gregory A Newby"

Akademický článek

Pioneer cells established by the [SWI+] prion can promote dispersal and out-crossing in yeast.

Publikováno v: PLoS Biology, Vol 15, Iss 11, p e2003476 (2017)

To thrive in an ever-changing environment, microbes must widely distribute their progeny to colonize new territory. Simultaneously, they must evolve and adapt to the stresses of unpredictable surroundings. In both of these regards, diversity is key-i

Externí odkaz: https://doaj.org/article/cf3d245720c2408fa8b4a3a01ab2d9f5

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Akademický článek

Precise correction of a spectrum of β-thalassemia mutations in coding and non-coding regions by base editors

Publikováno v: Molecular Therapy: Nucleic Acids, Vol 35, Iss 2, Pp 102205- (2024)

β-thalassemia/HbE results from mutations in the β-globin locus that impede the production of functional adult hemoglobin. Base editors (BEs) could facilitate the correction of the point mutations with minimal or no indel creation, but its efficienc

Externí odkaz: https://doaj.org/article/1c5f0f013cfe4f2da711f4e85aae51fa

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Akademický článek

Shuttle peptide delivers base editor RNPs to rhesus monkey airway epithelial cells in vivo

Publikováno v: Nature Communications, Vol 14, Iss 1, Pp 1-16 (2023)

Abstract Gene editing strategies for cystic fibrosis are challenged by the complex barrier properties of airway epithelia. We previously reported that the amphiphilic S10 shuttle peptide non-covalently combined with CRISPR-associated (Cas) ribonucleo

Externí odkaz: https://doaj.org/article/8ac54f1857804245a0069d9ef38b1f91

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Akademický článek

Protospacer modification improves base editing of a canonical splice site variant and recovery of CFTR function in human airway epithelial cells

Autor: Anya T. Joynt, Erin W. Kavanagh, Gregory A. Newby, Shakela Mitchell, Alice C. Eastman, Kathleen C. Paul, Alyssa D. Bowling, Derek L. Osorio, Christian A. Merlo, Shivani U. Patel, Karen S. Raraigh, David R. Liu, Neeraj Sharma, Garry R. Cutting

Publikováno v: Molecular Therapy: Nucleic Acids, Vol 33, Iss , Pp 335-350 (2023)

Canonical splice site variants affecting the 5′ GT and 3′ AG nucleotides of introns result in severe missplicing and account for about 10% of disease-causing genomic alterations. Treatment of such variants has proven challenging due to the unstab

Externí odkaz: https://doaj.org/article/bc5d8bbde920459b9b6b2f50f6c229eb

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Akademický článek

Reciprocal mutations of lung-tropic AAV capsids lead to improved transduction properties

Autor: Ashley L. Cooney, Christian M. Brommel, Soumba Traore, Gregory A. Newby, David R. Liu, Paul B. McCray, Patrick L. Sinn

Publikováno v: Frontiers in Genome Editing, Vol 5 (2023)

Considerable effort has been devoted to developing adeno-associated virus (AAV)-based vectors for gene therapy in cystic fibrosis (CF). As a result of directed evolution and capsid shuffling technology, AAV capsids are available with widespread tropi

Externí odkaz: https://doaj.org/article/9022961518b6498c9920e9f7bf41f264

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Akademický článek

Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy

Autor: Meha Kabra, Pawan K. Shahi, Yuyuan Wang, Divya Sinha, Allison Spillane, Gregory A. Newby, Shivani Saxena, Yao Tong, Yu Chang, Amr A. Abdeen, Kimberly L. Edwards, Cole O. Theisen, David R. Liu, David M. Gamm, Shaoqin Gong, Krishanu Saha, Bikash R. Pattnaik

Publikováno v: The Journal of Clinical Investigation, Vol 133, Iss 19 (2023)

Clinical genome editing is emerging for rare disease treatment, but one of the major limitations is the targeting of CRISPR editors’ delivery. We delivered base editors to the retinal pigmented epithelium (RPE) in the mouse eye using silica nanocap

Externí odkaz: https://doaj.org/article/3120d5bf8bdb4fc3874e0562564f1672

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Akademický článek

In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration

Autor: Elliot H. Choi, Susie Suh, Andrzej T. Foik, Henri Leinonen, Gregory A. Newby, Xin D. Gao, Samagya Banskota, Thanh Hoang, Samuel W. Du, Zhiqian Dong, Aditya Raguram, Sajeev Kohli, Seth Blackshaw, David C. Lyon, David R. Liu, Krzysztof Palczewski

Publikováno v: Nature Communications, Vol 13, Iss 1, Pp 1-14 (2022)

Leber congenital amaurosis is caused by mutations in RPE65 and leads to retinal degeneration in children. Here, the authors show that in vivo base editing can prolong the survival of cone photoreceptors and rescue their function in a mouse model of t

Externí odkaz: https://doaj.org/article/ffc071797e4f4fa694375352a34ae7c4

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Akademický článek

In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathies

Autor: Chang Li, Aphrodite Georgakopoulou, Gregory A. Newby, Kelcee A. Everette, Evangelos Nizamis, Kiriaki Paschoudi, Efthymia Vlachaki, Sucheol Gil, Anna K. Anderson, Theodore Koob, Lishan Huang, Hongjie Wang, Hans-Peter Kiem, David R. Liu, Evangelia Yannaki, André Lieber

Publikováno v: JCI Insight, Vol 7, Iss 19 (2022)

Individuals with β-thalassemia or sickle cell disease and hereditary persistence of fetal hemoglobin (HPFH) possessing 30% fetal hemoglobin (HbF) appear to be symptom free. Here, we used a nonintegrating HDAd5/35++ vector expressing a highly efficie

Externí odkaz: https://doaj.org/article/ad903bbc1db4441484b871ef2cf704a3

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