Rebecca Arens Nicholas
The physician who led the pioneering clinical work to develop and apply a custom CRISPR base editor therapy to treat a baby with a rare and fatal genetic disorder.
entitydetail.created_at
7/19/2025, 10:27:30 PM
entitydetail.last_updated
7/26/2025, 2:28:48 AM
entitydetail.research_retrieved
7/19/2025, 10:55:09 PM
Summary
Rebecca Arens Nicholas, also known as Rebecca Ahrens-Nicklas, is a physician with MD and PhD degrees, specializing in metabolic diseases and human genetics. She serves as an Attending Physician at Children's Hospital of Philadelphia, where she also directs the RASopathies Center, and is an Assistant Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania. Dr. Ahrens-Nicklas is recognized for her work in genetic disease treatment, notably for successfully treating a fatal genetic disease in an infant using advanced Base Editors, a breakthrough discussed on the All-In podcast.
Referenced in 1 Document
Research Data
Extracted Attributes
Award
Lysosomal WORLD Symposium Travel Award
Degrees
MD, PhD
Full Name
Rebecca Ahrens-Nicklas
Profession
Physician
Current Role
Attending Physician, Metabolic Disease Program and Division of Human Genetics
Certification
Pediatrics (American Board of Pediatrics)
Director Role
Director, RASopathies Center
Medical Degree
MD
Graduate Degree
PhD in Physiology & Biophysics
Current Affiliation
Children's Hospital of Philadelphia
Academic Affiliation
Perelman School of Medicine at the University of Pennsylvania
Academic Appointment
Assistant Professor of Pediatrics
Medical University (MD)
Weill Cornell Medical College, New York, NY
Graduate University (PhD)
Weill Cornell Medical College, New York, NY
Timeline
- Became a member of the American College of Medical Genetics and Genomics. (Source: web_search_results)
2014-01-01
- Became a member of the Society for the Study of Inborn Errors of Metabolism. (Source: web_search_results)
2014-01-01
- Became a member of the Society of Inborn Metabolic Disorders. (Source: web_search_results)
2014-01-01
- Became a member of the American Academy of Neurology. (Source: web_search_results)
2017-01-01
- Received the Lysosomal WORLD Symposium Travel Award. (Source: web_search_results)
2018-01-01
- Along with researchers from institutions like UCSF, successfully treated a fatal genetic disease in a baby using advanced Base Editors, a scientific breakthrough discussed on the All-In podcast. (Source: related_documents)
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Wikipedia
View on WikipediaLeslie Fox Prize for Numerical Analysis
The Leslie Fox Prize for Numerical Analysis of the Institute of Mathematics and its Applications (IMA) is a biennial prize established in 1985 by the IMA in honour of mathematician Leslie Fox (1918-1992). The prize honours "young numerical analysts worldwide" (any person who is less than 31 years old), and applicants submit papers for review. A committee reviews the papers, invites shortlisted candidates to give lectures at the Leslie Fox Prize meeting, and then awards First Prize and Second Prizes based on "mathematical and algorithmic brilliance in tandem with presentational skills."
Web Search Results
- Rebecca Ahrens-Nicklas, MD, PhD
## Secondary ## Main navigation ### Find a research study ### Innovation and your child ### Find research studies ## Sidebar ## Secondary ## Mobile Navigation # Rebecca Ahrens-Nicklas, MD, PhD ## Breadcrumb Rebecca Ahrens-Nicklas, MD, PhD ## Rebecca Ahrens-Nicklas, MD, PhD Rebecca Ahrens-Nicklas, MD, PhD, is an attending physician with the Metabolic Disease Program and the Division of Human Genetics at Children’s Hospital of Philadelphia. Locations: Main Building [...] ## About Rebecca Ahrens-Nicklas, MD, PhD ### Titles Attending Physician Director, RASopathies Center Assistant Professor of Pediatrics, Perelman School of Medicine at the University of Pennsylvania ### Certifications Clinical Biochemical Genetics – American Board of Medical Genetics and Genomics Clinical Genetics and Genomics – American Board of Medical Genetics and Genomics Pediatrics – American Board of Pediatrics ### Awards and Honors 2018, Lysosomal WORLD Symposium Travel Award [...] 2017-present, American Academy of Neurology 2014-present, American College of Medical Genetics and Genomics 2014-present, Society for the Study of Inborn Errors of Metabolism 2014-present, Society of Inborn Metabolic Disorders ### Education & training #### Graduate Degree PhD in Physiology & Biophysics - Weill Cornell Medical College, New York, NY #### Medical Degree MD - Weill Cornell Medical College, New York, NY #### Residency
- Current Students | Cell & Molecular Biology Graduate Group
Stephanie Blackwood (she/her/hers) ---------------------------------- ") Image 45: Stephanie Blackwood (she/her/hers) Gene Therapy and Vaccines Degree:PhD Matriculation Year: 2023 Advisor(s): Rebecca Ahrens-Nicklas Rotations: Kiran Musunuru Rebecca Ahrens-Nicklas Vijay Bhoj Louisa Boateng (she/her) ------------------------ ") Image 46: Louisa Boateng (she/her) Genetics and Epigenetics Degree:PhD Matriculation Year: 2022 [...] Home Students Current Students Current Students ================ Search Student Name, Description, Project Title, Advisor, or Rotations Filter Students by Program Filter Students by Degree Filter Students by Matriculation Year Filter Students by Advisor Reset Showing 1 to 50 of 451 matching students First Previous Page 1 Page 2 Page 3 Next Last = Website = Email No Results Found Amber Abbott ------------ Image 7: Amber Abbott [...] Current Students | Cell & Molecular Biology Graduate Group | Perelman School of Medicine at the University of Pennsylvania =============== Cell & Molecular Biology Graduate Group Close Menu) CAMB Home Open submenu (About)About Open submenu (Programs)Programs Open submenu (Students)Students Faculty Open submenu (Academics)Academics Admissions Open submenu (Resources)Resources Open submenu (News & Events)News & Events Student Newsletter
- Beta Gamma Sigma - Lutgert College of Business
Michele Sinito - [x] Michele Snyder - [x] Michelle Anderson - [x] Michelle Lynn Vastola - [x] Mickey Riley - [x] Miguel Garcia - [x] Miriam Pereira - [x] Mitchell Alexander Van Hamburg - [x] Molly Kate Fagan - [x] Monique Hashimoto - [x] Morgan Crawford - [x] Moses Chain - [x] Natalie Dravnel - [x] Natasha Cabernite - [x] Natasha Valdes - [x] Nathan Campbell - [x] Nathan M. Touchette - [x] Neel Chary - [x] Neva Cruz - [x] Nicholas Gallant - [x] Nicholas Irra - [x] Nicholas [...] Pedro Carle - [x] Pedro Lora - [x] Peter Dushku - [x] Peter Mitchell - [x] Petra Besenhard - [x] Petya Naydenova - [x] Petya Yaneva - [x] Philip Araujo - [x] Philipp Ruwe - [x] Prachi Ashar - [x] Preston Groenewold - [x] Quinn Starace - [x] Rachel Kanesha Coulanges - [x] Rachel Kogan - [x] Rachel McFalls - [x] Rachel Seifert - [x] Randy Hattab - [x] Reagan Scheck - [x] Rebecca S. Tyrrell - [x] Reese Harvey - [x] Renan Ueno - [x] Renee Danielle Faried - [x] Richard Lee [...] | Filter - [x] Aaron Scullion - [x] Aaron Selka - [x] Abbie Rose Sipos - [x] Abby N Schilling - [x] Adam Barreto - [x] Adam LeRoy Baack - [x] Adam Scigliano - [x] Ahmed El-Gendy - [x] Alan John Gurgal - [x] Alayna Salain - [x] Aldo Otarola - [x] Aleksandr Ulm - [x] Aleksandra Stankovska - [x] Alex Brown - [x] Alex Gambach - [x] Alex Philion - [x] Alex Scigliano - [x] Alexa Villalobos - [x] Alexander Gleason - [x] Alexandra Coburn - [x] Alexandra Danielle Dehen - [x] Alexey
- [PDF] English Language Arts and Literacy
approach informs the wording of Reading Standards 4 to 6, grouped together under the cluster heading Craft and Structure. By design, these standards are echoed in Language Standards 1 through 6, which deal with standard English conventions, language and style, and vocabulary development. 2. Comparative Analysis This approach is based on the concept that a reader gains understanding of a text by setting it in a broader context. This often means comparing it to other texts and seeking [...] understand how language functions in different contexts, to make effective choices for meaning or style, and to comprehend more fully when reading or listening. Vocabulary Acquisition and Use 4. Determine or clarify the meaning of unknown and multiple-meaning words and phrases by using context clues, analyzing meaningful word parts, and consulting general and specialized reference materials, as appropriate. 5. Demonstrate understanding of figurative language, word relationships, and nuances in [...] comprehend and evaluate complex texts across a range of types and disciplines, and they can construct effective arguments and convey intricate or multifaceted information. Likewise, students are able independently to discern a speaker’s key points, request clarification, and ask relevant questions. They build on others’ ideas, articulate their own ideas, and confirm they have been understood. Without prompting, they demonstrate command of standard English and acquire and use a wide-ranging
- Current clinical applications of AAV-mediated gene therapy - PubMed
that has thus far taken 3 decades to reduce to clinical practice, predominantly using adeno-associated viral (AAV) vectors. Among available viral and non-viral gene delivery approaches, AAV vectors remain the most efficient means for in vivo delivery of DNA to the nucleus. AAV vectors now constitute a bone fide novel therapeutic drug class composed of seven US Food and Drug Administration-approved products with over 10-fold more in clinical development for an expanding number of disease [...] ## Similar articles ## References ## Publication types ## MeSH terms ## Related information ## LinkOut - more resources ### Full Text Sources ### Medical Elsevier Science full text link NCBI Literature Resources MeSH PMC Bookshelf Disclaimer The PubMed wordmark and PubMed logo are registered trademarks of the U.S. Department of Health and Human Services (HHS). Unauthorized use of these marks is strictly prohibited. Connect with NLM [...] Currently, there are an estimated 8,000 genetic disorders that cumulatively affect approximately 10% of the population. Even among the 5% of patients with genetic disease that have treatment options, these therapeutics rarely address the underlying cause of disease but rather focus on managing or modifying symptoms and typically require recurrent, lifelong therapy. A therapeutic approach to genetic disease that in vivo delivers a functional copy of the aberrant gene is an intuitive solution