Results 41 to 50 of about 93,364 (279)

Clinical and Therapeutic Evaluation of the Ten Most Prevalent CRB1 Mutations

Biomedicines, 2023
Mutations in the Crumbs homolog 1 (CRB1) gene lead to severe inherited retinal dystrophies (IRDs), accounting for nearly 80,000 cases worldwide. To date, there is no therapeutic option for patients suffering from CRB1-IRDs.
Bruna Lopes da Costa, Masha Kolesnikova, Sarah R. Levi, Thiago Cabral, Stephen H. Tsang, Irene H. Maumenee, Peter M. J. Quinn   +6 more
doaj   +1 more source

In vivo somatic cell base editing and prime editing

Molecular Therapy, 2021
Recent advances in genome editing technologies have magnified the prospect of single-dose cures for many genetic diseases. For most genetic disorders, precise DNA correction is anticipated to best treat patients. To install desired DNA changes with high precision, our laboratory developed base editors (BEs), which can correct the four most common ...
Gregory A, Newby, David R, Liu
openaire   +2 more sources

Engineered pegRNAs improve prime editing efficiency

, 2022
Prime editing enables the installation of virtually any combination of point mutations, small insertions or small deletions in the DNA of living cells.
Chen, Jonathan, Hsu, Alvin, Randolph, Peyton B., Nelson, James W., An, Meirui, Shen, Simon, Newby, Gregory, Liu, David, Anzalone, Andrew, Chen, Peter, Everette, Kelcee A.   +10 more
core   +1 more source

A selectable all-in-one CRISPR prime editing piggyBac transposon allows for highly efficient gene editing in human cell lines

Scientific Reports, 2021
CRISPR prime-editors are emergent tools for genome editing and offer a versatile alternative approach to HDR-based genome engineering or DNA base-editors.
Reto Eggenschwiler, Thomas Gschwendtberger, Christian Felski, Christopher Jahn, Florian Langer, Jared Sterneckert, Andreas Hermann, Jonathan Lühmann, Doris Steinemann, Alexandra Haase, Ulrich Martin, Susanne Petri, Tobias Cantz   +12 more
doaj   +1 more source

A prime editing strategy to rewrite the γ-globin promoters and reactivate fetal hemoglobin for sickle cell disease.

Blood
International audienceFetal hemoglobin reactivation is a promising therapy for β-hemoglobinopathies. We developed a prime editing strategy that introduces multiple mutations in the fetal γ-globin promoters that are expected to increase their activity. We
Chalumeau A, Bou Dames M, Fontana L, Amistadi S, Antoniou P, Loganathan P, Mombled M, Corre G, Peterka M, Amendola M, Giovannangeli C, Maresca M, Miccio A, Brusson M.   +13 more
europepmc   +2 more sources

Predicting prime editing efficiency and product purity by deep learning

, 2023
Prime editing is a versatile genome editing tool but requires experimental optimization of the prime editing guide RNA (pegRNA) to achieve high editing efficiency.
Krauthammer, Michael; https://orcid.org/, Kissling, Lucas; https://orcid.org/, Schmidheini, Lukas; https://orcid.org/, Balázs, Zsolt, Allam, Ahmed; https://orcid.org/, Schwank, Gerald; https://orcid.org/, Mathis, Nicolas; https://orcid.org/, Solari, Cristina, Marquart, Kim Fabiano; https://orcid.org/   +8 more
core   +1 more source

Easy-Prime: a machine learning–based prime editor design tool

Genome Biology, 2021
Prime editing is a revolutionary genome-editing technology that can make a wide range of precise edits in DNA. However, designing highly efficient prime editors (PEs) remains challenging.
Yichao Li, Jingjing Chen, Shengdar Q. Tsai, Yong Cheng   +3 more
doaj   +1 more source

A versatile genetic engineering toolkit for E. coli based on CRISPR-prime editing

Nature Communications, 2021
CRISPR prime editing enables double-strand break free engineering of the genome. Here the authors present a toolkit for prime editing in E. coli.
Yaojun Tong, Tue S. Jørgensen, Christopher M. Whitford, Tilmann Weber, Sang Yup Lee   +4 more
doaj   +1 more source

Systematic optimization of prime editing for enhanced efficiency and versatility in genome engineering across diverse cell types

Frontiers in Cell and Developmental Biology
Prime editing offers remarkable versatility in genome editing, but its efficiency remains a major bottleneck. While continuous optimization of the prime editing enzymes and guide RNAs (pegRNAs) has improved editing outcomes, the method of delivery also ...
Huiling Mu, Huiling Mu, Yeyi Liu, Yeyi Liu, Yijia Chi, Yijia Chi, Fei Wang, Shuting Meng, Shuting Meng, Yi Zhang, Yi Zhang, Xunting Wang, Xunting Wang, Dongxin Zhao, Dongxin Zhao, Dongxin Zhao   +15 more
doaj   +1 more source

Multiplex base- and prime-editing with drive-and-process CRISPR arrays

Nature Communications, 2022
Current base- and prime-editing technologies lack efficient strategies to edit multiple genomic loci simultaneously, limiting their applications in complex genomics and polygenic diseases.
Qichen Yuan, Xue Gao
doaj   +1 more source