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Rebecca C. Ahrens‐Nicklas

University of Pennsylvania

23H-index
154Paper Count
1.7KCitation Count
Published Papers 80
Publication Date
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Evaluating cases of possible insertional mutagenesis after AAV gene therapy
errMolecular Therapy
IF12
err2026-07-23
err0
PREAI
errLindsey A. George; Rebecca C. Ahrens-Nicklas; Frederic D. Bushman
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Novel Features of RASopathies: Liver Disease as an Emerging Phenotype
errAmerican Journal of Medical Genetics Part A
IF1.7
err2026-02-01
err0
PREAI
errRippert, Alyssa L.; Strong, Alanna; Ahrens-nicklas, Rebecca C.
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errSave
Measurement and clinical interpretation of CRISPR off-targets
errNature Genetics
IF29
err2025-11-24
err0
PREAI
errAriella Angelini Stewart; Rebecca C. Ahrens-Nicklas; Shengdar Q. Tsai; Kiran Musunuru; Petros Giannikopoulos; Claire D. Clelland
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errSave
Base editing strategies for in-vivo correction of two highly recurrent phenylketonuria variants
errMolecular Therapy Nucleic Acids
IF6.1
err2025-11-10
err0
errOAAI
errAidan Quigley; Ishaan Jindal; Thomas Campion; Delaney Rutherford; Yongseok Han; Walsh Quigley; Ping Qu; Kiran Musunuru; Rebecca Ahrens-Nicklas; Xinying Hong; Xiao Wang
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errSave
How to create personalized gene editing platforms: Next steps toward interventional genetics
errThe American Journal of Human Genetics
IF0
err2025-10-31
err0
PREAI
errRebecca C. Ahrens-Nicklas; Kiran Musunuru
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errSave
Improved specificity and efficiency of in vivo adenine base editing therapies with hybrid guide RNAs
errNature Biomedical Engineering
IF26.6
err2025-10-28
err0
errOAAI
errMadelynn N. Whittaker; Lauren C. Testa; Aidan Quigley; Dominique L. Brooks; Sarah A. Grandinette; Hooda Said; Garima Dwivedi; Ishaan Jindal; Daphne Volpp; Julia L. Hacker; Ping Qu; Josh Zhiyong Wang; Michael A. Levine; Rebecca C. Ahrens-Nicklas; Qiaoli Li; Kiran Musunuru; Mohamad-Gabriel Alameh; William H. Peranteau; Xiao Wang
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Biallelic variants in BCAT1 impair mitochondrial function and are associated with a candidate neurometabolic disorder
errHuman Genetics and Genomics Advances
IF3.6
err2025-09-29
err0
errOAAI
errBrianna L. DiSanza; Giulia S. Porcari; Livia Sertori Finoti; Leonardo Ramos-Rodriguez; Devin M. Burris; Justin A. McDonough; Gang Ning; Grace Fagan; Guy T. Helman; Erin Weiss; Ryan J. Taft; Amy Pizzino; Matthew T. Whitehead; Amy Waldman; Cas Simons; Xilma Ortiz-Gonzalez; William C. Skarnes; Adeline Vanderver; Elizabeth J. Bhoj; Rebecca C. Ahrens-Nicklas
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errSave
Correction: CLN3 disease disrupts very early postnatal hippocampal maturation
errScientific Reports
IF3.9
err2025-08-18
err0
errOAAI
errJeet B. Singh; Devin M. Burris; Sangeetha Bhuyan; Tegan Thurston; Anna Oschmann; Connor Jankowski; Wenyun Lu; Joshua D. Rabinowitz; Rebecca C. Ahrens-Nicklas
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errSave
Pantethine ameliorates dilated cardiomyopathy features in PPCS deficiency disorder in patients and cell line models
errCommunications Medicine
IF6.3
err2025-07-31
err0
errOAAI
errFangfang Zhang; Tatjana Dorn; Barbara Gnutti; Yair Anikster; Sarah Kuebler; Rebecca Ahrens-Nicklas; Rachel Gosselin; Shamima Rahman; Ronen Durst; Enrica Zanuttigh; Miriam A. Güra; Christine M. Poch; Anna B. Meier; Karl-Ludwig Laugwitz; Hans-Joachim Schüller; Ana C. Messias; Ody C. Sibon; Dario Finazzi; Alyssa Rippert; Dong Li; Kristen Truxal; Deipanjan Nandi; Brent C. Lampert; Mildrid Yeo; Alice Gardham; Batel Nissan; Smadar Horowitz Cederboim; Alessandra Moretti; Arcangela Iuso
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errSave
Coupling deep phenotypic quantification with next-generation phenotyping for 192 individuals with germline histonopathies
errHUMAN GENETICS AND GENOMICS ADVANCES
IF3.6
err2025-07-01
err0
errOAAI
errLubin, Emily E.; Gonzalez, Elizabeth M.; Sangree, Annabel K.; Durham, Emily L.; Klinkhammer, Hannah; Li, Jing-Mei; Smith, Sarina M.; Layo-Carris, Dana E.; Clark, Kelly J.; Melendez-Perez, Ashley J.; Wang, Xiao Min; Angireddy, Rajesh; Weiss, Erin E.; Barakat, Tahsin Stefan; Mercier, Sandra; Cogne, Benjamin; Koene, Saskia; Hilhorst-Hofstee, Yvonne; Rydzanicz, Malgorzata; Ploski, Rafal; Cano, Maria de los Gomez; Palomares-Bralo, Maria; Arevalo, Tania Barragan; Tan, Tiong Yang; Gallacher, Lyndon; MacFarland, Suzanne P.; Ahrens-Nicklas, Rebecca C.; Nomakuchi, Tomoki T.; Bhoj, Elizabeth J. K.
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errSave
Reporting ABCD1 variants as actionable secondary findings on exome and genome sequencing
errGENETICS IN MEDICINE
IF6.2
err2025-07-01
err0
PREAI
errGonzalez, Carlos A. Dominguez; Spinner, Nancy B.; Ahrens-Nicklas, Rebecca C.; Young, Lisa R.; Voss, Laura A.; Reichert, Sara L.; Gallo, Daniel J.; Cohen, Julie S.; Bonkowsky, Joshua L.; Keller, Stephanie R.; Bennett, Mariko L.; Pizzino, Amy M.; Swantkowski, Meghan; Arnold, Kaley; Fraser, Jamie L.; Emerson, Felicity J.; Miettunen, Kelly; Fatemi, Ali; Haren, Keith P. Van; Adang, Laura; Waldman, Amy; Emrick, Lisa; Eichler, Florian; Vanderver, Adeline
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errSave
Longitudinal outcomes in Noonan syndrome
errGENETICS IN MEDICINE
IF6.2
err2025-04-01
err0
PREAI
errRippert, Alyssa L.; Reef, Rebecca; Mani, Ashika; Stefanatos, Arianna K.; Ahrens-Nicklas, Rebecca C.
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Functional connectivity changes in mouse models of maple syrup urine disease
errcortex
IF2.9
err2025-03-02
err0
PREAI
errLavery, Sarah; Adepoju, Temilola E.; Fisher, Hayden B.; Chan, Claudia; Kuhs, Amanda; Ahrens-Nicklas, Rebecca C.; White, Brian R.
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errSave
Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrations
errMOLECULAR THERAPY NUCLEIC ACIDS
IF6.1
err2025-03-01
err0
errOAAI
errTricoli, Lucas; Sase, Sunetra; Hacker, Julia L.; Pham, Vi; Chappell, Maxwell; Breda, Laura; Hurwitz, Stephanie N.; Tanaka, Naoto; Castracani, Carlo Castruccio; Guerra, Amaliris; Hou, Zhongqi; Schlotawa, Lars; Radhakrishnan, Karthikeyan; Hogenauer, Matthew; Roche, Aoife; Everett, John; Bushman, Frederic; Kurre, Peter; Ahrens-Nicklas, Rebecca; Adang, Laura A.; Vanderver, Adeline L.; Rivella, Stefano
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Five-Year Outcomes of Patients with Pompe Disease Identified by the Pennsylvania Newborn Screen
errINTERNATIONAL JOURNAL OF NEONATAL SCREENING
IF4
err2025-02-24
err0
errOAAI
errRon, Hayley A.; Kane, Owen; Guo, Rose; Menello, Caitlin; Engelhardt, Nicole; Pressley, Shaney; Diboscio, Brenda; Steffensen, Madeline; Cuddapah, Sanmati; Ng, Kim; Ficicioglu, Can; Ahrens-Nicklas, Rebecca C.
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errSave
Gene therapies for neurogenetic disorders
errTrends in Molecular Medicine
IF13.8
err2025-02-17
err0
errOAAI
errOrrin Devinsky; Jeff Coller; Rebecca Ahrens-Nicklas; X. Shawn Liu; Nadav Ahituv; Beverly L. Davidson; Kathie M. Bishop; Yael Weiss; Ana Mingorance
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errSave
Contribution of Brain Intrinsic Branched-Chain Amino Acid Metabolism in a Novel Mouse Model of Maple Syrup Urine Disease
errJOURNAL OF INHERITED METABOLIC DISEASE
IF3.8
err2025-02-04
err1
errOAAI
errKuhs, Amanda C.; Ohl, Laura; Thurston, Tegan; Singh, Jeet; Bhuyan, Sangeetha; Grandinette, Sarah; Xu, Jing; Siemsgluess, Sophie A.; Jakher, Youseff; Ahrens-Nicklas, Rebecca C.
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Bone marrow transplantation reverses metabolic alterations in multiple sulfatase deficiency: a case series
errCOMMUNICATIONS MEDICINE
IF6.3
err2025-01-09
err0
errOAAI
errPillai, Nishitha R.; Liu, Ning; Li, Xiyuan; Li, Xiqi; Ahrens-Nicklas, Rebecca; Adang, Laura; Eisengart, Julie B.; Bronken, Grace; Gupta, Ashish; Lund, Troy C.; Whitley, Chester B.; Elsea, Sarah H.; Orchard, Paul J.
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Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency
errMOLECULAR THERAPY
IF12
err2024-11-01
err1
errOAAI
errPham, Vi; Tricoli, Lucas; Hong, Xinying; Wongkittichote, Parith; Castracani, Carlo Castruccio; Guerra, Amaliris; Schlotawa, Lars; Adang, Laura A.; Kuhs, Amanda; Cassidy, Margaret M.; Kane, Owen; Tsai, Emily; Presa, Maximiliano; Lutz, Cathleen; Rivella, Stefano B.; Ahrens-Nicklas, Rebecca C.
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Bone marrow transplantation increases sulfatase activity in somatic tissues in a multiple sulfatase deficiency mouse model
errCOMMUNICATIONS MEDICINE
IF6.3
err2024-10-25
err0
errOAAI
errPresa, Maximiliano; Pham, Vi; Ray, Somdatta; Piec, Pierre-Alexandre; Ryan, Jennifer; Billings, Timothy; Coombs, Harold; Schlotawa, Lars; Lund, Troy; Ahrens-Nicklas, Rebecca C.; Lutz, Cathleen
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Research Directions

No research directions
Co-authors
Cooperation Journals
T
Ted M. Dawson
H-index: 160 · Papers: 710
V
Valina L. Dawson
H-index: 151 · Papers: 579
F
Frederic D. Bushman
H-index: 139 · Papers: 781
J
Joshua D. Rabinowitz
H-index: 127 · Papers: 769
O
Orrin Devinsky
H-index: 121 · Papers: 1.2K
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