Most cited papers from Regenxbio (United States)

Regenxbio (United States)

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

The most cited paper from Regenxbio (United States) is "Anifrolumab, an Anti–Interferon‐α Receptor Monoclonal Antibody, in Moderate‐to‐Severe Systemic Lupus Erythematosus" (Richard Alan Furie, Munther A. Khamashta, Joan T. Merrill et al., 2016), with 884 citations. Next come "Preclinical and clinical evaluation of the LRRK2 inhibitor DNL201 for Parkinson’s disease" (266) and "AAV8-vectored suprachoroidal gene transfer produces widespread ocular transgene expression" (141).

Published TypeFieldAccessCitations

Showing 50 of 61 papers. Every paper with more than 11 citations is listed.

Most cited papers, sortable by citations and release date
#	Paper
1	Anifrolumab, an Anti–Interferon‐α Receptor Monoclonal Antibody, in Moderate‐to‐Severe Systemic Lupus Erythematosus Richard Alan Furie, Munther A. Khamashta, Joan T. Merrill et al. · Arthritis & Rheumatology Open access	7 Nov 2016	884
2	Preclinical and clinical evaluation of the LRRK2 inhibitor DNL201 for Parkinson’s disease Danna L. Jennings et al. · Science Translational Medicine	8 Jun 2022	266
3	AAV8-vectored suprachoroidal gene transfer produces widespread ocular transgene expression Kun Ding, Jikui Shen, Zibran Hafiz et al. · Journal of Clinical Investigation Open access	13 Aug 2019	141
4	Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study Peter A. Campochiaro, Robert L. Avery, David M. Brown et al. · The Lancet	27 Mar 2024	129
5	Addressing the Value of Gene Therapy and Enhancing Patient Access to Transformative Treatments Rachel Salzman, Francesca Cook, Timothy D. Hunt et al. · Molecular Therapy Open access	29 Oct 2018	115
6	2019 White Paper on Recent Issues in Bioanalysis: FDA Immunogenicity Guidance, Gene Therapy, Critical Reagents, Biomarkers and Flow Cytometry Validation (Part 3 – Recommendations on 2019 FDA Immunogenicity Guidance, Gene Therapy Bioanalytical Challenges, Strategies for Critical Reagent Management, Biomarker Assay Validation, Flow Cytometry Validation & CLSI H62) Steven P. Piccoli et al. · Bioanalysis Open access	Dec 2019	97
7	Characterization of AAV-mediated dorsal root ganglionopathy Nicholas A. P. S. Buss, Lisa G. Lanigan, Jillynne Zeller et al. · Molecular Therapy — Methods & Clinical Development Open access	Feb 2022	80
8	Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I Lalitha R. Belur, Alexa Temme, Kelly M. Podetz-Pedersen et al. · Human Gene Therapy Open access	2 May 2017	67
9	Disruption of NOTCH signaling by a small molecule inhibitor of the transcription factor RBPJ Cecilia Hurtado, A. G. Safarova, Michael Peter Smith et al. · Scientific Reports Open access	25 Jul 2019	60
10	Recommendations for the Development of Cell-Based Anti-Viral Vector Neutralizing Antibody Assays Boris Gorovits, Michele Fiscella, Mike Havert et al. · The AAPS Journal	6 Jan 2020	58
11	AAV8-antiVEGFfab Ocular Gene Transfer for Neovascular Age-Related Macular Degeneration Yuanyuan Liu, Seth D. Fortmann, Jikui Shen et al. · Molecular Therapy Open access	8 Dec 2017	55
12	Stability and Dissociation of Adeno-Associated Viral Capsids by Variable Temperature-Charge Detection-Mass Spectrometry Marius M. Kostelic, Jack P. Ryan, Levi S. Brown et al. · Analytical Chemistry	18 Aug 2022	49
13	Application of Bayesian methods to accelerate rare disease drug development: scopes and hurdles Kelley M. Kidwell, Satrajit Roychoudhury, Barbara A. Wendelberger et al. · Orphanet Journal of Rare Diseases Open access	7 May 2022	48
14	Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System–Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer Kanut Laoharawee, Kelly M. Podetz-Pedersen, Tam T. T. N. Nguyen et al. · Human Gene Therapy	6 May 2017	48
15	Recommendations for Method Development and Validation of qPCR and dPCR Assays in Support of Cell and Gene Therapy Drug Development Amanda L. Hays et al. · The AAPS Journal Open access	5 Feb 2024	47
16	Evaluation of Cellular Immune Response to Adeno-Associated Virus-Based Gene Therapy Boris Gorovits, Mitra Azadeh, George Buchlis et al. · The AAPS Journal Open access	26 Apr 2023	46
17	Principles for interactions with biopharmaceutical companies: the development of guidelines for patient advocacy organizations in the field of rare diseases Susan M. Stein, Elizabeth Bogard, Nicole Boice et al. · Orphanet Journal of Rare Diseases Open access	22 Jan 2018	45
18	Recombinant human B cell repertoires enable screening for rare, specific, and natively paired antibodies Saravanan Rajan, Michael R. Kierny, Andrew A. Mercer et al. · Communications Biology Open access	16 Jan 2018	45
19	Kinetics and durability of transgene expression after intrastriatal injection of AAV9 vectors Bradley S. Hollidge, Hayley B. Carroll, Randolph Qian et al. · Frontiers in Neurology Open access	14 Nov 2022	44
20	Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I Lalitha R. Belur, Megan Romero, Junggu Lee et al. · Frontiers in Molecular Neuroscience Open access	10 May 2021	44
21	Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice Christian J. Hinderer, Nathan Katz, Jean‐Pierre Louboutin et al. · Human Gene Therapy	10 Aug 2016	44
22	UniDecCD: Deconvolution of Charge Detection-Mass Spectrometry Data Marius M. Kostelic, Ciara K. Zak, Yang Liu et al. · Analytical Chemistry Open access	27 Oct 2021	43
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	Engineering of the CMV promoter for controlled expression of recombinant genes in HEK293 cells Yusuf B. Johari, Joseph M. Scarrott, Thilo H. Pohle et al. · Biotechnology Journal Open access	28 Apr 2022	37
25	Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials Jeffrey S. Chamberlain, Melissa A. Robb, Serge Braun et al. · Human Gene Therapy Open access	25 Jan 2023	34
26	Analytical characterization of coformulated antibodies as combination therapy Jun Kim, Yoen Joo Kim, Mingyan Cao et al. · mAbs Open access	2020	34
27	Gene therapy using an ortholog of human fragile X mental retardation protein partially rescues behavioral abnormalities and EEG activity Alexander W.M. Hooper, Hayes Wong, Yosuke Niibori et al. · Molecular Therapy — Methods & Clinical Development Open access	16 Jul 2021	33
28	Liver-Targeted AAV8 Gene Therapy Ameliorates Skeletal and Cardiovascular Pathology in a Mucopolysaccharidosis IVA Murine Model Kazuki Sawamoto, Subha Karumuthil‐Melethil, Shaukat Ali Khan et al. · Molecular Therapy — Methods & Clinical Development Open access	21 May 2020	33
29	GluK2 Is a Target for Gene Therapy in Drug‐Resistant Temporal Lobe Epilepsy Céline Boileau et al. · Annals of Neurology Open access	21 Jun 2023	31
30	Sexually dimorphic patterns in electroencephalography power spectrum and autism-related behaviors in a rat model of fragile X syndrome Hayes Wong, Alexander W.M. Hooper, Yosuke Niibori et al. · Neurobiology of Disease Open access	5 Oct 2020	31
31	Prevalence of Anti–Adeno-Associated Virus Serotype 8 Neutralizing Antibodies and Arylsulfatase B Cross-Reactive Immunologic Material in Mucopolysaccharidosis VI Patient Candidates for a Gene Therapy Trial Rita Ferla, Pamela Claudiani, Marco Savarese et al. · Human Gene Therapy Open access	5 Feb 2015	31
32	Neutralizing Antibody Validation Testing and Reporting Harmonization Heather A. Myler et al. · The AAPS Journal Open access	8 Jul 2023	30
33	Sex Difference Leads to Differential Gene Expression Patterns and Therapeutic Efficacy in Mucopolysaccharidosis IVA Murine Model Receiving AAV8 Gene Therapy Matthew Piechnik, Paige C. Amendum, Kazuki Sawamoto et al. · International Journal of Molecular Sciences Open access	21 Oct 2022	30
34	Increased recombinant adeno‐associated virus production by HEK293 cells using small molecule chemical additives Joseph M. Scarrott, Yusuf B. Johari, Thilo H. Pohle et al. · Biotechnology Journal Open access	10 Dec 2022	29
35	Toll-like receptor 4 mediates blood-brain barrier permeability and disease in C3H mice during Venezuelan equine encephalitis virus infection Bradley S. Hollidge, Courtney A. Cohen, Justice Akuoku Frimpong et al. · Virulence Open access	25 Jan 2021	29
36	Quantitation of Trace Levels of DNA Released from Disrupted Adeno-Associated Virus Gene Therapy Vectors Jared S. Bee, Kristin O'Berry, Yu Zhang et al. · Journal of Pharmaceutical Sciences Open access	6 Jun 2021	28
37	Liver-Directed Adeno-Associated Virus–Mediated Gene Therapy for Mucopolysaccharidosis Type VI Nicola Brunetti‐Pierri et al. · NEJM Evidence Open access	6 Jun 2022	27
38	Respiratory syncytial virus fusion nanoparticle vaccine immune responses target multiple neutralizing epitopes that contribute to protection against wild-type and palivizumab-resistant mutant virus challenge Brian E. Gilbert, Nita Patel, Hanxin Lu et al. · Vaccine	31 Oct 2018	27
39	Medicaid coverage practices for approved gene and cell therapies: Existing barriers and proposed policy solutions Jeremy G. Allen, Diane C. Berry, Francesca Cook et al. · Molecular Therapy — Methods & Clinical Development Open access	17 May 2023	26
40	Generation of reference cell lines, media, and a process platform for CHO cell biomanufacturing Lauren T. Cordova et al. · Biotechnology and Bioengineering	22 Nov 2022	25
41	Impact of Time Out of Intended Storage and Freeze-thaw Rates on the Stability of Adeno-associated Virus 8 and 9 Jared S. Bee, Yu Zhang, Megan Kuhn Phillippi et al. · Journal of Pharmaceutical Sciences Open access	6 Jan 2022	25
42	Development of a stable lyophilized adeno-associated virus gene therapy formulation Yu Zhang, Roberto A. DePaz, Jared S. Bee et al. · International Journal of Pharmaceutics	21 Jul 2021	25
43	Symmetric Age Association of Retinal Degeneration in Patients with CLN2-Associated Batten Disease Kyle D. Kovacs, Samir C. Patel, Anton Orlin et al. · Ophthalmology Retina Open access	21 Jan 2020	25
44	An online survey of burden of illness in families with mucopolysaccharidosis type II children in the United States Therese Conner, Francesca Cook, Vivian Fernandez et al. · Molecular Genetics and Metabolism Reports Open access	30 Aug 2019	25
45	Mechanistic Studies and Formulation Mitigations of Adeno-associated Virus Capsid Rupture During Freezing and Thawing Jared S. Bee, Yu Zhang, Sheyla Finkner et al. · Journal of Pharmaceutical Sciences	26 Mar 2022	24
46	2023 White Paper on Recent Issues in Bioanalysis: ISR for ADA Assays, the Rise of dPCR vs qPCR, International Reference Standards for Vaccine Assays, Anti-AAV TAb Post-Dose Assessment, NanoString Validation, ELISpot as Gold Standard (Part 3 – Recommendations on Gene Therapy, Cell Therapy, Vaccines Immunogenicity & Technologies; Biotherapeutics Immunogenicity & Risk Assessment; ADA/NAb Assay/Report Johanna R. Mora et al. · Bioanalysis Open access	22 Feb 2024	21
47	Community consensus for Heparan sulfate as a biomarker to support accelerated approval in Neuronopathic Mucopolysaccharidoses Joseph Muenzer, Carole Ho, Heather Lau et al. · Molecular Genetics and Metabolism Open access	10 Jul 2024	20
48	Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model Keigo Takahashi, Elizabeth M. Eultgen, Sophie H. Wang et al. · Journal of Clinical Investigation Open access	27 Apr 2023	19
49	Design of synthetic promoters for controlled expression of therapeutic genes in retinal pigment epithelial cells Yusuf B. Johari, Andrew C. Mercer, Ye Liu et al. · Biotechnology and Bioengineering Open access	12 Feb 2021	19
50	Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10 Lalitha R. Belur, Kelly M. Podetz-Pedersen, Thuy Tran et al. · Molecular Genetics and Metabolism Reports Open access	20 May 2020	19

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.