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Michael C. Jaskolka

Gene Editing for CEP290 -Associated Retinal Degeneration
2024 · Eric A. Pierce, Tomas S. Aleman, et al. · New England Journal of Medicine · 268
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A highly efficient transgene knock-in technology in clinically relevant cell types
2023 · Alexander G. Allen, Samia Q. Khan, et al. · Nature Biotechnology · 71
RAVE and Rabconnectin-3 Complexes as Signal Dependent Regulators of Organelle Acidification
2021 · Michael C. Jaskolka, Samuel R. Winkley, et al. · Frontiers in Cell and Developmental Biology · 49
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Coordinated conformational changes in the V1 complex during V-ATPase reversible dissociation
2022 · Thamiya Vasanthakumar, Kristine A. Keon, et al. · Nature Structural & Molecular Biology · 36
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CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat Sickle Cell Disease
2026 · Rabi Hanna, Haydar Frangoul, et al. · New England Journal of Medicine · 29
CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat β-Thalassemia
2026 · Haydar Frangoul, Rabi Hanna, et al. · New England Journal of Medicine · 26
Defining steps in RAVE-catalyzed V-ATPase assembly using purified RAVE and V-ATPase subcomplexes
2021 · Michael C. Jaskolka, Maureen Tarsio, et al. · Journal of Biological Chemistry · 24
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Interaction between the yeast RAVE complex and Vph1-containing Vo sectors is a central glucose-sensitive interaction required for V-ATPase reassembly
2020 · Michael C. Jaskolka, Patricia M. Kane · Journal of Biological Chemistry · 21
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AsCas12a Gene Editing of HBG1/2 Promoters with EDIT-301 Results in Rapid and Sustained Normalization of Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia
2023 · Rabi Hanna, Haydar Frangoul, et al. · Blood · 20
S264: Edit-301 Shows Promising Preliminary Safety and Efficacy Results in the Phase I/ii Clinical Trial (ruby) of Patients With Severe Sickle Cell Disease Using Highly Specific and Efficient Ascas12a Enzyme
2023 · Rabi Hanna, Haydar Frangoul, et al. · HemaSphere · 20
Artemisia vulgaris L. Chemotypes
2013 · Jack D. Williams, Michael A. Campbell, et al. · American Journal of Plant Sciences · 11
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AsCas12a Gene Editing of HBG1/2 Promoters with Edit-301 Results in Early and Sustained Normalization of Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia
2024 · Haydar Frangoul, Rabi Hanna, et al. · Transplantation and Cellular Therapy · 8
Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Successful Engraftment, Increased Hemoglobin, and Reduced Transfusion Dependence in Patients with Transfusion-Dependent Beta-Thalassemia Treated in the Edithal Trial
2024 · Haydar Frangoul, Rabi Hanna, et al. · Blood · 5
CRISPR-Cas12a gene editing of the HBG1/2 promoters leads to sustained normalization of total hemoglobin and increased fetal hemoglobin in patients with severe sickle cell disease: Updated Results from the RUBY trial
2025 · Rabi Hanna, Haydar Frangoul, et al. · Blood · 3
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Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Sustained Hemoglobin Normalization and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease Treated in the RUBY Trial
2025 · Mark Walters, Haydar Frangoul, et al. · Transplantation and Cellular Therapy · 2
Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Successful Engraftment, Increased Hemoglobin, and Reduced Transfusion Dependence in Patients with Transfusion-Dependent Beta-Thalassemia Treated in the Edithal Trial
2025 · Haydar Frangoul, Rabi Hanna, et al. · Transplantation and Cellular Therapy · 2
Abstract 4364195: A transformative LDL cholesterol–lowering in vivo CRISPR gene editing medicine that functionally upregulates LDLR in mice and non-human primates
2025 · Judith Newmark, Paul Wrighton, et al. · Circulation · 1
The Human Rogdi Protein Is the Functional Homologue of Yeast Rav2 and Can Promote V‐ATPase Assembly
2021 · Samuel Winkley, Michael Jaskolka, et al. · The FASEB Journal · 1
Deciphering the isoform code contained in V o a‐subunit isoforms of the V‐ATPase
2020 · Patricia M Kane, Michael C. Jaskolka, et al. · The FASEB Journal · 1
A Transformative in Vivo Crispr Gene Editing Medicine Upregulates Ldlr and Meaningfully Reduces Ldl-c in Non-human Primates
2026 · Paul Wrighton, Judith Newmark, et al. · Atherosclerosis
Ergonomic determinants of neutral posture in outpatient dermatologic surgery: A literature review
2026 · Phuong Daniels, Michelle Tashjian, et al. · JAAD reviews.
A Transformative In Vivo CRISPR Gene Editing Medicine Leads to Upregulation of LDLR and Robust Reduction of Two Independent ASCVD Risk Factors, LDL-C and Lp(a), in Preclinical Studies
2026 · Paul Wrighton, Judith Newmark, et al. · Journal of clinical lipidology
Some reassembly required: Requirements for RAVE‐mediated reassembly of the yeast V‐ATPase
2019 · Michael C Jaskolka, Patricia Kane · The FASEB Journal
Works count: 23
Citation count: 598
H-index: 11
I10-index: 11
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