Search Results - "Newby, Gregory A."

Efficient C•G-to-G•C base editors developed using CRISPRi screens, target-library analysis, and machine learning by Koblan, Luke W., Arbab, Mandana, Shen, Max W., Hussmann, Jeffrey A., Anzalone, Andrew V., Doman, Jordan L., Newby, Gregory A., Yang, Dian, Mok, Beverly, Replogle, Joseph M., Xu, Albert, Sisley, Tyler A., Weissman, Jonathan S., Adamson, Britt, Liu, David R.

“…Programmable C•G-to-G•C base editors (CGBEs) have broad scientific and therapeutic potential, but their editing outcomes have proved difficult to predict and…”
Get full text

Functional correction of CFTR mutations in human airway epithelial cells using adenine base editors by Krishnamurthy, Sateesh, Traore, Soumba, Cooney, Ashley L, Brommel, Christian M, Kulhankova, Katarina, Sinn, Patrick L, Newby, Gregory A, Liu, David R, McCray, Paul B

“…Mutations in the CFTR gene that lead to premature stop codons or splicing defects cause cystic fibrosis (CF) and are not amenable to treatment by…”
Get full text

Cross-Kingdom Chemical Communication Drives a Heritable, Mutually Beneficial Prion-Based Transformation of Metabolism by Jarosz, Daniel F., Brown, Jessica C.S., Walker, Gordon A., Datta, Manoshi S., Ung, W. Lloyd, Lancaster, Alex K., Rotem, Assaf, Chang, Amelia, Newby, Gregory A., Weitz, David A., Bisson, Linda F., Lindquist, Susan

“…In experimental science, organisms are usually studied in isolation, but in the wild, they compete and cooperate in complex communities. We report a system for…”
Get full text

Base Editor Correction of COL7A1 in Recessive Dystrophic Epidermolysis Bullosa Patient-Derived Fibroblasts and iPSCs by Osborn, Mark J., Newby, Gregory A., McElroy, Amber N., Knipping, Friederike, Nielsen, Sarah C., Riddle, Megan J., Xia, Lily, Chen, Weili, Eide, Cindy R., Webber, Beau R., Wandall, Hans H., Dabelsteen, Sally, Blazar, Bruce R., Liu, David R., Tolar, Jakub

“…Genome editing represents a promising strategy for the therapeutic correction of COL7A1 mutations that cause recessive dystrophic epidermolysis bullosa (RDEB)…”
Get full text

Pioneer cells established by the [SWI+] prion can promote dispersal and out-crossing in yeast by Newby, Gregory A, Lindquist, Susan

“…To thrive in an ever-changing environment, microbes must widely distribute their progeny to colonize new territory. Simultaneously, they must evolve and adapt…”
Get full text

Chemical modifications of adenine base editor mRNA and guide RNA expand its application scope by Jiang, Tingting, Henderson, Jordana M., Coote, Kevin, Cheng, Yi, Valley, Hillary C., Zhang, Xiao-Ou, Wang, Qin, Rhym, Luke H., Cao, Yueying, Newby, Gregory A., Bihler, Hermann, Mense, Martin, Weng, Zhiping, Anderson, Daniel G., McCaffrey, Anton P., Liu, David R., Xue, Wen

“…CRISPR-Cas9-associated base editing is a promising tool to correct pathogenic single nucleotide mutations in research or therapeutic settings. Efficient base…”
Get full text

Prime editing in mice reveals the essentiality of a single base in driving tissue-specific gene expression by Gao, Pan, Lyu, Qing, Ghanam, Amr R, Lazzarotto, Cicera R, Newby, Gregory A, Zhang, Wei, Choi, Mihyun, Slivano, Orazio J, Holden, Kevin, Walker, 2nd, John A, Kadina, Anastasia P, Munroe, Rob J, Abratte, Christian M, Schimenti, John C, Liu, David R, Tsai, Shengdar Q, Long, Xiaochun, Miano, Joseph M

“…Most single nucleotide variants (SNVs) occur in noncoding sequence where millions of transcription factor binding sites (TFBS) reside. Here, a comparative…”
Get full text

In vivo base editing rescues cone photoreceptors in a mouse model of early-onset inherited retinal degeneration by Choi, Elliot H., Suh, Susie, Foik, Andrzej T., Leinonen, Henri, Newby, Gregory A., Gao, Xin D., Banskota, Samagya, Hoang, Thanh, Du, Samuel W., Dong, Zhiqian, Raguram, Aditya, Kohli, Sajeev, Blackshaw, Seth, Lyon, David C., Liu, David R., Palczewski, Krzysztof

“…Leber congenital amaurosis (LCA) is the most common cause of inherited retinal degeneration in children. LCA patients with RPE65 mutations show accelerated…”
Get full text

Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo by Zhang, Han, Kelly, Karen, Lee, Jonathan, Echeverria, Dimas, Cooper, David, Panwala, Rebecca, Amrani, Nadia, Chen, Zexiang, Gaston, Nicholas, Wagh, Atish, Newby, Gregory A, Xie, Jun, Liu, David R, Gao, Guangping, Wolfe, Scot A, Khvorova, Anastasia, Watts, Jonathan K, Sontheimer, Erik J

“…Abstract Guide RNAs offer programmability for CRISPR-Cas9 genome editing but also add challenges for delivery. Chemical modification, which has been key to the…”
Get full text

A Genetic Tool to Track Protein Aggregates and Control Prion Inheritance by Newby, Gregory A., Kiriakov, Szilvia, Hallacli, Erinc, Kayatekin, Can, Tsvetkov, Peter, Mancuso, Christopher P., Bonner, J. Maeve, Hesse, William R., Chakrabortee, Sohini, Manogaran, Anita L., Liebman, Susan W., Lindquist, Susan, Khalil, Ahmad S.

“…Protein aggregation is a hallmark of many diseases but also underlies a wide range of positive cellular functions. This phenomenon has been difficult to study…”
Get full text

Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy by Kabra, Meha, Shahi, Pawan K, Wang, Yuyuan, Sinha, Divya, Spillane, Allison, Newby, Gregory A, Saxena, Shivani, Tong, Yao, Chang, Yu, Abdeen, Amr A, Edwards, Kimberly L, Theisen, Cole O, Liu, David R, Gamm, David M, Gong, Shaoqin, Saha, Krishanu, Pattnaik, Bikash R

“…Clinical genome editing is emerging for rare disease treatment, but one of the major limitations is the targeting of CRISPR editors' delivery. We delivered…”
Get full text

Shuttle peptide delivers base editor RNPs to rhesus monkey airway epithelial cells in vivo by Kulhankova, Katarina, Traore, Soumba, Cheng, Xue, Benk-Fortin, Hadrien, Hallée, Stéphanie, Harvey, Mario, Roberge, Joannie, Couture, Frédéric, Kohli, Sajeev, Gross, Thomas J., Meyerholz, David K., Rettig, Garrett R., Thommandru, Bernice, Kurgan, Gavin, Wohlford-Lenane, Christine, Hartigan-O’Connor, Dennis J., Yates, Bradley P., Newby, Gregory A., Liu, David R., Tarantal, Alice F., Guay, David, McCray, Paul B.

“…Gene editing strategies for cystic fibrosis are challenged by the complex barrier properties of airway epithelia. We previously reported that the amphiphilic…”
Get full text

Amphiphilic shuttle peptide delivers base editor ribonucleoprotein to correct the CFTR R553X mutation in well-differentiated airway epithelial cells by Kulhankova, Katarina, Cheng, Anna X, Traore, Soumba, Auger, Maud, Pelletier, Mia, Hervault, Maxime, Wells, Kevin D, Green, Jonathan A, Byrne, Addison, Nelson, Benjamin, Sponchiado, Mariana, Boosani, Chandra, Heffner, Caleb S, Snow, Kathy J, Murray, Stephen A, Villacreses, Raul A, Rector, Michael V, Gansemer, Nicholas D, Stoltz, David A, Allamargot, Chantal, Couture, Frédéric, Hemez, Colin, Hallée, Stéphanie, Barbeau, Xavier, Harvey, Mario, Lauvaux, Coraline, Gaillet, Bruno, Newby, Gregory A, Liu, David R, McCray, Jr, Paul B, Guay, David

“…Base editing could correct nonsense mutations that cause cystic fibrosis (CF), but clinical development is limited by the lack of delivery methods that…”
Get full text

In vivo somatic cell base editing and prime editing by Newby, Gregory A., Liu, David R.

“…Recent advances in genome editing technologies have magnified the prospect of single-dose cures for many genetic diseases. For most genetic disorders, precise…”
Get full text

Protospacer modification improves base editing of a canonical splice site variant and recovery of CFTR function in human airway epithelial cells by Joynt, Anya T., Kavanagh, Erin W., Newby, Gregory A., Mitchell, Shakela, Eastman, Alice C., Paul, Kathleen C., Bowling, Alyssa D., Osorio, Derek L., Merlo, Christian A., Patel, Shivani U., Raraigh, Karen S., Liu, David R., Sharma, Neeraj, Cutting, Garry R.

“…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…”
Get full text

Precise correction of a spectrum of β-thalassemia mutations in coding and non-coding regions by base editors by Prasad, Kirti, Devaraju, Nivedhitha, George, Anila, Ravi, Nithin Sam, Paul, Joshua, Mahalingam, Gokulnath, Rajendiran, Vignesh, Panigrahi, Lokesh, Venkatesan, Vigneshwaran, Lakhotiya, Kartik, Periyasami, Yogapriya, Pai, Aswin Anand, Nakamura, Yukio, Kurita, Ryo, Balasubramanian, Poonkuzhali, Thangavel, Saravanabhavan, Velayudhan, Shaji R., Newby, Gregory A., Marepally, Srujan, Srivastava, Alok, Mohankumar, Kumarasamypet M.

“…β-thalassemia/HbE results from mutations in the β-globin locus that impede the production of functional adult hemoglobin. Base editors (BEs) could facilitate…”
Get full text

In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathies by Li, Chang, Georgakopoulou, Aphrodite, Newby, Gregory A, Everette, Kelcee A, Nizamis, Evangelos, Paschoudi, Kiriaki, Vlachaki, Efthymia, Gil, Sucheol, Anderson, Anna K, Koob, Theodore, Huang, Lishan, Wang, Hongjie, Kiem, Hans-Peter, Liu, David R, Yannaki, Evangelia, Lieber, André

“…Individuals with β-thalassemia or sickle cell disease and hereditary persistence of fetal hemoglobin (HPFH) possessing 30% fetal hemoglobin (HbF) appear to be…”
Get full text

Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors by Doman, Jordan L., Raguram, Aditya, Newby, Gregory A., Liu, David R.

“…Cytosine base editors (CBEs) enable targeted C•G-to-T•A conversions in genomic DNA. Recent studies report that BE3, the original CBE, induces a low frequency…”
Get full text

Precision genome editing using cytosine and adenine base editors in mammalian cells by Huang, Tony P., Newby, Gregory A., Liu, David R.

“…Genome editing has transformed the life sciences and has exciting prospects for use in treating genetic diseases. Our laboratory developed base editing to…”
Get full text

Reciprocal mutations of lung-tropic AAV capsids lead to improved transduction properties by Cooney, Ashley L, Brommel, Christian M, Traore, Soumba, Newby, Gregory A, Liu, David R, McCray, Jr, Paul B, Sinn, Patrick L

“…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…”
Get full text