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R. Chandler · SciRah

Researcher profile · Semantic Scholar

R. Chandler

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R. Chandler — researcher profile on SciRah

h-index: 29
Works: 74
Citations: 2,989

Most-cited papers

Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy.
R. Chandler, M. LaFave, G. Varshney, N. Trivedi +7 more
2015Journal of Clinical Investigation361 citations Open access
Semantic Scholar
Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia
D. An, Jessica L. Schneller, A. Frassetto, Shi Liang +20 more
2017Cell Reports231 citations Open access
Semantic Scholar
Combined methylmalonic acidemia and homocystinuria, cblC type. I. Clinical presentations, diagnosis and management
Nuria Carrillo-Carrasco, R. Chandler, C. Venditti
2011Journal of Inherited Metabolic Disease221 citations Open access
Semantic Scholar
Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype.
R. Sarkar, R. Tetreault, G. Gao, Lili Wang +4 more
2004Blood211 citations Open access
Semantic Scholar
Mitochondrial dysfunction in mut methylmalonic acidemia
R. Chandler, P. Zerfas, S. Shanske, J. Sloan +3 more
2009The FASEB Journal191 citations Open access
Semantic Scholar
Evaluating the state of the science for adeno-associated virus integration: An integrated perspective
D. Sabatino, Frederic D. Bushman, R. Chandler, R. Crystal +10 more
2022Molecular Therapy180 citations Open access
Semantic Scholar
Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models
R. Chandler, M. Sands, C. Venditti
2017Human Gene Therapy176 citations Open access
Semantic Scholar
Targeting proximal tubule mitochondrial dysfunction attenuates the renal disease of methylmalonic acidemia
I. Manoli, J. Sysol, Lingli Li, P. Houillier +19 more
2013Proceedings of the National Academy of Sciences of the United States of America115 citations Open access
Semantic Scholar
Exome sequencing identifies ACSF3 as the cause of Combined Malonic and Methylmalonic Aciduria
J. Sloan, J. Johnston, I. Manoli, R. Chandler +20 more
2011Nature Genetics105 citations Open access
Semantic Scholar
Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1
R. Chandler, Ian M. Williams, Alana L. Gibson, Cristin D. Davidson +5 more
2016Human Molecular Genetics68 citations Open access
Semantic Scholar
FGF21 underlies a hormetic response to metabolic stress in methylmalonic acidemia.
I. Manoli, J. Sysol, M. Epping, Li-Na Li +27 more
2018JCI Insight66 citations Open access
Semantic Scholar
Metabolic phenotype of methylmalonic acidemia in mice and humans: the role of skeletal muscle
R. Chandler, J. Sloan, H. Fu, Matthew S Tsai +5 more
2007BMC Medical Genetics62 citations Open access
Semantic Scholar
Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy?
R. Chandler, M. LaFave, G. Varshney, S. Burgess +1 more
2016Molecular Therapy57 citations Open access
Semantic Scholar
Liver-Directed Recombinant Adeno-Associated Viral Gene Delivery Rescues a Lethal Mouse Model of Methylmalonic Acidemia and Provides Long-Term Phenotypic Correction
Nuria Carrillo-Carrasco, R. Chandler, Suma Chandrasekaran, C. Venditti
2010Human Gene Therapy56 citations Open access
Semantic Scholar
Genetic and genomic systems to study methylmalonic acidemia.
R. Chandler, C. Venditti
2005Molecular Genetics and Metabolism55 citations Open access
Semantic Scholar
Promoterless, Nuclease‐Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia
R. Chandler, L. Venturoni, Jing Liao, Brandon T. Hubbard +10 more
2020Hepatology49 citations Open access
Semantic Scholar
Adeno-Associated Virus Serotype 8 Gene Transfer Rescues a Neonatal Lethal Murine Model of Propionic Acidemia
R. Chandler, Suma Chandrasekaran, Nuria Carrillo-Carrasco, J. Senac +3 more
2011Human Gene Therapy49 citations Open access
Semantic Scholar
Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future
R. Chandler, C. Venditti
2019Human Gene Therapy48 citations Open access
Semantic Scholar
Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors
R. Ferla, M. Alliegro, Margherita Dell’Anno, E. Nusco +13 more
2020Molecular Therapy: Methods & Clinical Development47 citations Open access
Semantic Scholar
Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe disease.
Yedda Li, Christopher A. Miller, L. Shea, Xun-Tian Jiang +9 more
2020Molecular Therapy44 citations Open access
Semantic Scholar
Long-term Rescue of a Lethal Murine Model of Methylmalonic Acidemia Using Adeno associated Viral Gene Therapy
R. Chandler, C. Venditti
2009Molecular Therapy40 citations Open access
Semantic Scholar
Adenovirus-Mediated Gene Delivery Rescues a Neonatal Lethal Murine Model of mut 0 Methylmalonic Acidemia
R. Chandler, C. Venditti
2008Human Gene Therapy37 citations Open access
Semantic Scholar
Liver-Directed Adeno-Associated Virus Serotype 8 Gene Transfer Rescues a Lethal Murine Model of Citrullinemia Type 1
R. Chandler, T. Tarasenko, K. Cusmano-Ozog, Qin Sun +3 more
2013Gene Therapy36 citations Open access
Semantic Scholar
The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup
P. Brooks, E. Ottinger, D. Portero, Richa Madan Lomash +35 more
2020Human Gene Therapy33 citations Open access
Semantic Scholar
Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia
Elizabeth A Harrington, J. Sloan, I. Manoli, R. Chandler +5 more
2016Human Gene Therapy33 citations Open access
Semantic Scholar
ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia
P. Ilyinskii, Alicia M. Michaud, G. Rizzo, C. Roy +12 more
2021Molecular Therapy: Methods & Clinical Development32 citations Open access
Semantic Scholar
Propionyl-CoA and adenosylcobalamin metabolism in Caenorhabditis elegans: evidence for a role of methylmalonyl-CoA epimerase in intermediary metabolism.
R. Chandler, V. Aswani, Matthew S Tsai, Marni J. Falk +6 more
2006Molecular Genetics and Metabolism32 citations Open access
Semantic Scholar
The proteome of methylmalonic acidemia (MMA): elucidation of altered pathways in patient livers
M. Caterino, R. Chandler, J. Sloan, K. Dorko +7 more
2016Molecular Biosystems31 citations Open access
Semantic Scholar
Ion-abrasion scanning electron microscopy reveals distorted liver mitochondrial morphology in murine methylmalonic acidemia
G. Murphy, Bradley C. Lowekamp, P. Zerfas, R. Chandler +3 more
2010Journal of Structural Biology30 citations Open access
Semantic Scholar
Gene therapy in a murine model of Methylmalonic Acidemia (MMA) using rAAV9 mediated gene delivery
J. Senac, R. Chandler, J. Sysol, L. Li +1 more
2011Gene Therapy29 citations Open access
Semantic Scholar
Gene therapy for metabolic diseases
R. Chandler, C. Venditti
2016Translational Science of Rare Diseases27 citations Open access
Semantic Scholar
Pre-clinical efficacy and dosing of an AAV8 vector expressing human methylmalonyl-CoA mutase in a murine model of methylmalonic acidemia (MMA)
R. Chandler, C. Venditti
2012Molecular Genetics and Metabolism27 citations Open access
Semantic Scholar
Adenoviral-mediated correction of methylmalonyl-CoA mutase deficiency in murine fibroblasts and human hepatocytes
R. Chandler, Matthew S Tsai, K. Dorko, J. Sloan +4 more
2007BMC Medical Genetics22 citations Open access
Semantic Scholar
Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.
D. An, Jessica L. Schneller, A. Frassetto, Shi Liang +20 more
2018Cell Reports21 citations Open access
Semantic Scholar
Gene therapy for organic acidemias: Lessons learned from methylmalonic and propionic acidemia
R. Chandler, C. Venditti
2023Journal of Inherited Metabolic Disease19 citations
Semantic Scholar
Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann–Pick disease type C1 mice
Cristin D. Davidson, Alana L. Gibson, Tansy Gu, Laura L. Baxter +11 more
2021Life Science Alliance18 citations Open access
Semantic Scholar
Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease
H. Kang, Monika Gjorgjieva, Stephanie N. Smith, E. Brooks +10 more
2019Molecular Therapy: Methods & Clinical Development18 citations Open access
Semantic Scholar
In vivo genome editing at the albumin locus to treat methylmalonic acidemia
Jessica L. Schneller, Ciaran M. Lee, L. Venturoni, R. Chandler +7 more
2021Molecular Therapy: Methods & Clinical Development16 citations Open access
Semantic Scholar
Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9
R. Chandler, G. Di Pasquale, J. Sloan, Samantha McCoy +5 more
2022Molecular Therapy: Methods & Clinical Development14 citations Open access
Semantic Scholar
Studies on protozoa. Part II: The golden hamster (Cricetus auratus) and cotton rat (Sigmodon hispidus) as experimental hosts for Leishmania donovani.
J. Fulton, L. Joyner, R. Chandler
1950Transactions of the Royal Society of Tropical Medicine and Hygiene14 citations
Semantic Scholar
Messenger RNA therapy as an option for treating metabolic disorders
R. Chandler
2019Proceedings of the National Academy of Sciences of the United States of America13 citations Open access
Semantic Scholar
Growth advantage of corrected hepatocytes in a juvenile model of methylmalonic acidemia following liver directed adeno-associated viral mediated nuclease-free genome editing
L. Venturoni, R. Chandler, Jing Liao, V. Hoffmann +4 more
2022Molecular Genetics and Metabolism10 citations Open access
Semantic Scholar
Systemic gene therapy using an AAV44.9 vector rescues a neonatal lethal mouse model of propionic acidemia
R. Chandler, G. Di Pasquale, Eun-Young Choi, David Chang +6 more
2023Molecular Therapy: Methods & Clinical Development9 citations Open access
Semantic Scholar
Long-term Rescue of a Lethal Murine Model of Methylmalonic Acidemia Using Adeno associated Viral Gene Therapy.
R. Chandler, C. Venditti
2010Molecular Therapy8 citations Open access
Semantic Scholar
Systemic AAV 9 gene therapy improves the lifespan of mice with Niemann-Pick disease , type C 1
R. Chandler, Ian M. Williams, Alana L. Gibson, Cristin D. Davidson +5 more
20167 citations
Semantic Scholar
Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program
Richa Madan Lomash, Jean Dehdashti, Oleg A. Shchelochkov, R. Chandler +13 more
2025Human Gene Therapy6 citations Open access
Semantic Scholar
Dissecting the effect of mitochondrial BCAT inhibition in methylmalonic acidemia
Madeline Hemmingsen, Guo-Fang Zhang, Yunhan Ma, Hannah Marchuk +16 more
2025JCI Insight5 citations Open access
Semantic Scholar
Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemia
Francis J. May, PamelaSara E. Head, L. Venturoni, R. Chandler +1 more
2021Molecular Therapy: Methods & Clinical Development5 citations Open access
Semantic Scholar
A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates.
R. Chandler, C. Venditti
2011Molecular Therapy4 citations Open access
Semantic Scholar
Adeno-Associated Viral Gene Therapy Using PHP.B:NPC1 Ameliorates Disease Phenotype in Mouse Model of Niemann-Pick C1 Disease
Alana L. Gibson, Cristin D. Davidson, R. Chandler, B. Deverman +5 more
20173 citations
Semantic Scholar

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C. Venditti×45
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J. Sloan×12
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V. Hoffmann×7
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V. Hoffmann — researcher profile on SciRah
I. Manoli×7
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I. Manoli — researcher profile on SciRah

Nuria Carrillo-Carrasco

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Li-Na Li — researcher profile on SciRah

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S. Burgess — researcher profile on SciRah

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P. Zerfas — researcher profile on SciRah

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J. Sysol — researcher profile on SciRah

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K. Cusmano-Ozog — researcher profile on SciRah

Alana L. Gibson×4

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Alana L. Gibson — researcher profile on SciRah