Matthew McNeill

Integrated DNA Technologies (United States)

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G. Lelais

Genomics Institute of the Novartis Research Foundation

Pierre‐Yves Michellys

Genomics Institute of the Novartis Research Foundation

Robert Epple

German Center for Neurodegenerative Diseases

Thomas H. Marsilje

Genomics Institute of the Novartis Research Foundation

Mari Manuia

Genomics Institute of the Novartis Research Foundation

Michael DiDonato

Genomics Institute of the Novartis Research Foundation

Badry Bursulaya

Novartis (United States)

Shailaja Kasibhatla

Boundless Bio (United States)

Garrett R. Rettig

Integrated DNA Technologies (United States)

Mark A. Behlke

Integrated DNA Technologies (United States)

Cooperative Institutions

Genomics Institute of the Novartis Research Foundation

The University of Texas MD Anderson Cancer Center

Harvard University

University of Chicago

University of Divinity

Yale University

Colgate Rochester Crozer Divinity School

Dana-Farber Cancer Institute

Integrated DNA Technologies (United States)

University of Iowa

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Identitet och invandring : en kvalitativ studie med ensamkommande flyktingbarn

Jennie Larsson Matthew McNeill

Identity and immigration - A qualitative study with unaccompanied refugee children Jennie ...

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CHH volume 22 issue 2 Cover and Front matter

Church History (1953)

Philip Schaff F T H E Legislature Of N EBASCO SERVICES INC NEW YORK Sidney E. Mead Mervin M. Deems

An abstract is not available for this content so a preview has been provided. As you have access to this content, a full PDF is available via the ‘Save PDF’ action button.

Front cover

Content (measure theory)

10.1017/s0009640700005084

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A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

Nature Medicine (2018)

Christopher A. Vakulskas Daniel P. Dever Garrett R. Rettig Rolf Turk Ashley M. Jacobi

10.1038/s41591-018-0137-0

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Supplementary Figure Legends from EGF816 Exerts Anticancer Effects in Non–Small Cell Lung Cancer by Irreversibly and Selectively Targeting Primary and Acquired Activating Mutations in the EGF Receptor

Yong Jia José Juárez Jie Li Mari Manuia Matthew J. Niederst

<p>This file contains the supplementary figure legends. Figure S1 shows the chemical structure of EGF816 and its binding to EGFR T790M mutant. Figure S2 shows the tolerability of EGF816 in the in vivo xenograft models. Figure S3 shows the in vivo PK and PD after a single dose of EGF816. Figure S4 shows the selectivity profile of EGF816 in the kinome tree. Figure S5 shows the morphology of EGF816 resistance clones.</p>

T790M

Kinome

Tolerability

10.1158/0008-5472.22409409

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CHH volume 22 issue 3 Cover and Front matter

Church History (1953)

Philip Schaff F T H E Legislature Of N EBASCO SERVICES INC NEW YORK Sidney E. Mead Mervin M. Deems

An abstract is not available for this content so a preview has been provided. As you have access to this content, a full PDF is available via the ‘Save PDF’ action button.

Front cover

Content (measure theory)

10.1017/s0009640700005230

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Novel tricyclic pyrazolopyrimidines as potent and selective GPR119 agonists

Bioorganic & Medicinal Chemistry Letters (2014)

Mihai Azimioara Phil B. Alper Christopher Cow Daniel Mutnick V. I. Nikulin

Tricyclic

10.1016/j.bmcl.2014.10.010

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High-level correction of the sickle mutation amplified in vivo during erythroid differentiation

bioRxiv (Cold Spring Harbor Laboratory) (2018)

Wendy Magis Mark A. DeWitt Stacia K. Wyman Jonathan T. Vu Seok‐Jin Heo

ABSTRACT Sickle Cell Disease (SCD), one of the world’s most common genetic disorders, causes anemia and progressive multiorgan damage that typically shortens lifespan by decades; currently there is no broadly applicable curative therapy. Here we show that Cas9 RNP-mediated gene editing with an ssDNA oligonucleotide donor yields markerless correction of the sickle mutation in more than 30% of long-term engrafting human hematopoietic stem cells (HSCs), using a selection-free protocol that is directly applicable to a clinical setting. We further find that in vivo erythroid differentiation markedly enriches for corrected ß-globin alleles. Adoption of a high-fidelity Cas9 variant demonstrates that this approach can yield efficient editing with almost no off-target events. These findings indicate that the sickle mutation can be corrected in human HSCs at curative levels with a streamlined protocol that is ready to be translated into a therapy. ONE SENTENCE SUMMARY Cas9-mediated correction of the sickle mutation in human hematopoietic stem cells can be accomplished at curative levels.

10.1101/432716

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In vivo selection for corrected β-globin alleles after CRISPR/Cas9 editing in human sickle hematopoietic stem cells enhances therapeutic potential

Wendy Magis Mark A. DeWitt Stacia K. Wyman Jonathan T. Vu Seok‐Jin Heo

Sickle Cell Disease (SCD), one of the world9s most common genetic disorders, causes anemia and progressive multiorgan damage that typically shortens lifespan by decades; currently there is no broadly applicable curative therapy. Here we show that Cas9 RNP-mediated gene editing with an ssDNA oligonucleotide donor yields more than 20% correction of the sickle mutation in long-term engrafting human HSCs. Using RNA-seq, we further find that in vivo erythroid differentiation markedly enriches for cells carrying corrected β-globin alleles. Adoption of a high-fidelity Cas9 variant demonstrates that this approach can yield efficient editing with almost no off-target events. These findings indicate that the sickle mutation can be corrected in human HSCs at levels that are likely to be curative if translated into a therapy.

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CRISPAltRations: A validated cloud-based approach for interrogation of double-strand break repair mediated by CRISPR genome editing

Molecular Therapy — Methods & Clinical Development (2021)

Gavin Kurgan Rolf Turk Heng Li Nathan Roberts Garrett R. Rettig

10.1016/j.omtm.2021.03.024

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Citations (23)

Supplementary Table 1 from EGF816 Exerts Anticancer Effects in Non–Small Cell Lung Cancer by Irreversibly and Selectively Targeting Primary and Acquired Activating Mutations in the EGF Receptor

Yong Jia José Juárez Jie Li Mari Manuia Matthew J. Niederst

<p>This file contains the supplementary Table S1 that describes the characteristics and anti-proliferative activities of EGF816 on a number of patient-derived cell lines</p>

Table (database)

10.1158/0008-5472.22409400

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