Agilent Technologies, Inc.

Camp4 Therapeutics Corporation

Subhadeep Roy

Daniel E Ryan

Douglas J Dellinger

David A Bumcrot

Gusti Zeiner

Jeffrey Robert Sampson

Alfica Sehgal

Chong Wing Yung

Andrew Kennedy

Yun Joon Jung

Yuchun Guo

Bryan J Matthews

Yuting Liu

Rachana S Kelkar

Mario Esteban Contreras Gamboa

Rutuja Sudhakar Pai

Described herein are methods of modulating gene transcription using antisense oligonucleotides (ASOs) targeting regulatory RNAs, such as promoter-associated RNAs and enhancer RNAs. These methods are useful for modulating the levels of gene products, for example, increasing expression of Carbamoyl-Phosphatase Synthetase 1 (CPS1), thereby treating diseases associated with aberrant gene expression.

Modulation of gene transcription using antisense oligonucleotides targeting regulatory RNAs

The present invention relates to guide RNAs having chemical modifications and their use in CRISPR-Cas systems. The chemically modified guide RNAs have enhanced specificity for target polynucleotide sequences. The present invention also relates to methods of using chemically modified guide RNAs for cleaving or nicking polynucleotides, and for high specificity genome editing.

High specificity genome editing using chemically modified guide RNAs

The present invention relates to guide RNAs comprising adaptor segments having one or more modifications, and their use in homologous recombination by CRISPR:Cas systems. The modified adaptor segments are resistant to degradation by RNaseH. The present invention also relates to a dual guide RNA strategy in which a first guide RNA directs a Cas enzyme to make a double-strand break at a first target sequence, and a second guide RNA comprises an adaptor segment attached to a donor polynucleotide, and binds a second target sequence that is offset from the first target sequence.

Compounds and methods for CRISPR/Cas-based genome editing by homologous recombination

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Lafayette, CO, United States of America

Subhadeep Roy