Most cited papers from AskBio (United States)

AskBio (United States)

What are the most cited papers from AskBio (United States)?

The most cited paper from AskBio (United States) is "Dystrophin Immunity in Duchenne's Muscular Dystrophy" (Jerry R. Mendell, Katherine Campbell, Louise R. Rodino‐Klapac et al., 2010), with 668 citations. Next come "Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector" (399) and "AAV vectors: The Rubik’s cube of human gene therapy" (377).

Published TypeFieldAccessCitations

Showing 50 of 72 papers. Every paper is listed.

Most cited papers, sortable by citations and release date
#	Paper
1	Dystrophin Immunity in Duchenne's Muscular Dystrophy Jerry R. Mendell, Katherine Campbell, Louise R. Rodino‐Klapac et al. · New England Journal of Medicine Open access	6 Oct 2010	668
2	Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector Dawn E. Bowles, Scott McPhee, Chengwen Li et al. · Molecular Therapy Open access	8 Nov 2011	399
3	AAV vectors: The Rubik’s cube of human gene therapy Amaury Pupo, Audry Fernández, Siew Hui Low et al. · Molecular Therapy Open access	5 Oct 2022	377
4	Long-Term Follow-Up After Gene Therapy for Canavan Disease Paola Leone, David M. Shera, Scott McPhee et al. · Science Translational Medicine Open access	19 Dec 2012	330
5	Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle Aravind Asokan, Julia C Conway, Jana L. Phillips et al. · Nature Biotechnology Open access	27 Dec 2009	271
6	BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression Barbara A. Konkle, Christopher Walsh, Miguel Antonio Escobar et al. · Blood Open access	17 Oct 2020	190
7	Immunogenicity assessment of AAV-based gene therapies: An IQ consortium industry white paper Tong‐Yuan Yang, Manuela Braun, Wibke Lembke et al. · Molecular Therapy — Methods & Clinical Development Open access	19 Aug 2022	127
8	Employing a Gain-of-Function Factor IX Variant R338L to Advance the Efficacy and Safety of Hemophilia B Human Gene Therapy: Preclinical Evaluation Supporting an Ongoing Adeno-Associated Virus Clinical Trial Paul Edward Monahan, Junjiang Sun, Tong Gui et al. · Human Gene Therapy Open access	24 Nov 2014	109
9	Phase I Trial of Autologous RNA-electroporated cMET-directed CAR T Cells Administered Intravenously in Patients with Melanoma and Breast Carcinoma Payal Deepak Shah et al. · Cancer Research Communications Open access	3 Apr 2023	102
10	Hematopoietic stem cell transplantation chemotherapy causes microglia senescence and peripheral macrophage engraftment in the brain Kurt A. Sailor, Georgios Agoranos, Sergio López-Manzaneda et al. · Nature Medicine Open access	21 Feb 2022	90
11	Cardiac I-1c Overexpression With Reengineered AAV Improves Cardiac Function in Swine Ischemic Heart Failure Kiyotake Ishikawa, Kenneth Michael Fish, Lisa M. Tilemann et al. · Molecular Therapy Open access	15 Jul 2014	86
12	Long-term safety of MRI-guided administration of AAV2-GDNF and gadoteridol in the putamen of individuals with Parkinson’s disease Matthew T. Rocco, Asad S. Akhter, Debra J. Ehrlich et al. · Molecular Therapy Open access	10 Aug 2022	63
13	Self-complementary AAV2.5-BMP2-coated Femoral Allografts Mediated Superior Bone Healing Versus Live Autografts in Mice With Equivalent Biomechanics to Unfractured Femur Cemal Yazıcı, Masahiko Takahata, David G. Reynolds et al. · Molecular Therapy Open access	4 Jan 2011	57
14	Adeno-Associated Virus Capsid-Promoter Interactions in the Brain Translate from Rat to the Nonhuman Primate Martin O. Bohlen, Thomas J. McCown, Sara Powell et al. · Human Gene Therapy Open access	17 Sep 2020	48
15	Protein Phosphatase Inhibitor-1 Gene Therapy in a Swine Model of Nonischemic Heart Failure Shin Watanabe, Kiyotake Ishikawa, Kenneth Michael Fish et al. · Journal of the American College of Cardiology Open access	25 Sep 2017	47
16	Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer Chengwen Li, Shuqing Wu, Blake H. Albright et al. · Molecular Therapy Open access	29 Jul 2015	47
17	An assessment of prognostic immunity markers in breast cancer Benlong Yang, J.-H Chou, Yaozhong Tao et al. · npj Breast Cancer Open access	25 Oct 2018	46
18	Phase I study of liver depot gene therapy in late-onset Pompe disease Edward C. Smith, Sam Hopkins, Laura E. Case et al. · Molecular Therapy Open access	19 Feb 2023	45
19	Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents Holly Landrum Peay, Ryan A. Fischer, Janice Tzeng et al. · PLoS ONE Open access	May 2019	45
20	Prediction of adeno-associated virus neutralizing antibody activity for clinical application Mian Wang, Ashley J. Crosby, Eric L. Hastie et al. · Gene Therapy Open access	30 Jun 2015	45
21	An Update on Gene Therapy Approaches for Parkinson’s Disease: Restoration of Dopaminergic Function Amber D. Van Laar, Victor S. Van Laar, Waldy San Sebastián et al. · Journal of Parkinson s Disease Open access	6 Aug 2021	44
22	Preclinical biodistribution, tropism, and efficacy of oligotropic AAV/Olig001 in a mouse model of congenital white matter disease Jeremy S. Francis, Vladimir Markov, Irenuez D. Wojtas et al. · Molecular Therapy — Methods & Clinical Development Open access	21 Jan 2021	41
23	2021 White Paper on Recent Issues in Bioanalysis: TAb/NAb, Viral Vector CDx, Shedding Assays; CRISPR/Cas9 & CAR-T Immunogenicity; PCR & Vaccine Assay Performance; ADA Assay Comparability & Cut Point Appropriateness ( Part 3 – Recommendations on Gene Therapy, Cell Therapy, Vaccine Assays; Immunogenicity of Biotherapeutics and Novel Modalities; Integrated Summary of Immunogenicity Harmonization) LiNa Loo et al. · Bioanalysis Open access	17 May 2022	37
24	Intraputaminal Delivery of Adeno‐Associated Virus Serotype 2–Glial Cell Line–Derived Neurotrophic Factor in Mild or Moderate Parkinson's Disease Amber D. Van Laar, Chadwick W. Christine, Nicolás M. Phielipp et al. · Movement Disorders Open access	20 May 2025	33
25	Gene Therapy in Movement Disorders: A Systematic Review of Ongoing and Completed Clinical Trials Aristide Merola, Noelle Kobayashi, Alberto Romagnolo et al. · Frontiers in Neurology ReviewOpen access	6 Apr 2021	29
26	Cardiotropic AAV gene therapy for heart failure: a phase 1 trial Timothy D. Henry et al. · Nature Medicine Open access	21 Oct 2025	28
27	Efficient transduction of vascular smooth muscle cells with a translational AAV2.5 vector: a new perspective for in-stent restenosis gene therapy A-M Lompré, Lahouaria Hadri, E Merlet et al. · Gene Therapy Open access	28 Mar 2013	28
28	PK/PD and Bioanalytical Considerations of AAV-Based Gene Therapies: an IQ Consortium Industry Position Paper Uma Kavita et al. · The AAPS Journal	31 Jul 2023	21
29	Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD Britt Erika Hanson, Sofia Stenler, Nina Ahlskog et al. · Molecular Therapy — Nucleic Acids Open access	23 Oct 2022	21
30	Adeno-associated virus for the treatment of muscle diseases: toward clinical trials. Nina DiPrimio, Scott McPhee, Richard Jude Samulski · PubMed	Oct 2010	21
31	Strategies for enhanced gene delivery to the central nervous system Zhenghong Gao · Nanoscale Advances Open access	2024	19
32	Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model Audrey Bourdon et al. · Gene Therapy Open access	Feb 2022	19
33	The effect of surface demineralization of cortical bone allograft on the properties of recombinant adeno-associated virus coatings Cemal Yazıcı, Laura Yanoso, Chao Lian Xie et al. · Biomaterials Open access	Jul 2008	17
34	Thermostability and in vivo performance of AAV9 in a film matrix Trang Nguyen Kieu Doan, Matthew D. Le, Irnela Bajrovic et al. · Communications Medicine Open access	21 Nov 2022	15
35	2024 White Paper on Recent Issues in Bioanalysis: Evolution of Immunogenicity Assessment beyond ADA/NAb; Regulated Genomic/NGS Assays; Hypersensitivity Reactions; Minimum Noise Reduction; False Positive Range; Modernized Vaccine Approaches; NAb/TAb Correlation (PART 3A – Recommendations on Advanced Strategies for Molecular Assays and Immunogenicity of Gene Therapy, Cell Therapy, Vaccine; Biotherap Omar Tounekti et al. · Bioanalysis Open access	25 Jan 2025	11
36	Phase 1b Safety and Preliminary Efficacy of Bilateral Intraputaminal Delivery of AAV2 GDNF (AB-1005) in Participants With Mild or Moderate Parkinson’s Disease (N2.001) Amber D. Van Laar, Chadwick W. Christine, Aristide Merola et al. · Neurology	9 Apr 2024	10
37	Gene therapy for cross-correction of somatic organs and the CNS in mucopolysaccharidosis II in rodents and non-human primates Nancy Chen, David E. Ehmann, Robert Crooker et al. · Molecular Therapy — Methods & Clinical Development Open access	28 Mar 2023	10
38	AAV-HDV: An Attractive Platform for the In Vivo Study of HDV Biology and the Mechanism of Disease Pathogenesis Sheila Maestro, Nahia Gómez-Echarte, Gracián Camps et al. · Viruses Open access	28 Apr 2021	10
39	ASPIRO Gene Therapy Trial In X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Safety And Efficacy Findings up to 72 Weeks Post-Treatment (1053) Perry B. Shieh et al. · Neurology	14 Apr 2020	10
40	Biobetter Biologics Alexandra Beumer Sassi, Radhika P. Nagarkar, P. F. Hamblin · Elsevier eBooks Book chapter	2015	10
41	Non-canonical role for the ataxia-telangiectasia-Rad3 pathway in STAT3 activation in human multiple myeloma cells Lin Li, Xiaoyan Hu, Jewel Nkwocha et al. · Cellular Oncology Open access	May 2023	9
42	Direct Comparison of Epifluorescence and Immunostaining for Assessing Viral Mediated Gene Expression in the Primate Brain Tierney B. Daw, Hala G. El-Nahal, Michele A. Basso et al. · Human Gene Therapy Open access	31 Jan 2023	8
43	Src inhibition potentiates MCL-1 antagonist activity in acute myeloid leukemia Xiaoyan Hu, Lin Li, Jewel Nkwocha et al. · Signal Transduction and Targeted Therapy Open access	9 Feb 2025	7
44	FAST principles for preprint feedback Sandra Franco-Iborra, Jessica Polka, Sara E. Monaco et al. PreprintOpen access	13 Jan 2022	7
45	Pharmacotherapy of cardiovascular diseases from herbs and pills to nucleic acids Ulf Landmesser et al. · European Heart Journal Open access	21 Aug 2025	4
46	Assessment of Hippocampal Adeno-Associated Viral Vector Gene Delivery via Frameless Stereotaxis in a Nonhuman Primate Cary H. Leung, Michele Ann Kliem, Brenten L. Heeke et al. · Stereotactic and Functional Neurosurgery Open access	2011	4
47	Gene therapy for cardiomyocytes, a heart beat away Scott McPhee, R J Samulski · Gene Therapy	30 Apr 2009	4
48	2025 White Paper on Recent Issues in Bioanalysis: Biomarkers Calibrators & Stability; Evaluation of NULISA; Neurofilament & Autoantibody Biomarker Assays; Removing IgM Interference; ELISpot & FluoroSpot Best Practices; Modular HD Cytometry; Single-cell Analysis Imaging Cytometry (PART 2A – Recommendations on Biomarkers Discovery, Development, Validation & Regulatory Approval, Ligand-Binding Assays Sarah Hersey et al. · Bioanalysis Open access	2 Dec 2025	3
49	Astrocyte-neuron combined targeting for CYP46A1 gene therapy in Huntington’s disease Louis-Habib Parsai, Farah Chali, Enejda Subashi et al. · Acta Neuropathologica Communications Open access	26 Aug 2025	3
50	GenePHIT Phase 2 Study Design: A Double Blind, Placebo-controlled Trial To Assess Efficacy, Safety, And Tolerability Of Ab-1002 Gene Therapy In Adults With Heart Failure Timothy D. Henry, Eugene S. Chung, Monica Alvisi et al. · Journal of Cardiac Failure	2025	3

Citations are OpenAlex's own counts, from its 2026-09-23 snapshot, so the ranking can differ from Google Scholar or Web of Science. A paper is listed for an institution when any of its authors gave it as an affiliation; retracted papers and papers whose citation count cannot be traced to citing works are left out, and a preprint and the article it became are shown once. Release dates are OpenAlex's: where a source gave only a month or a year, that is what is shown. How the lists are chosen.