Randy J. Chandler

National Human Genome Research Institute

Biochemistry, Genetics and Molecular BiologyMedicine

Pilot corpus only

This score is computed over theSindex pilot corpus and does not cover the full scientific literature. Scores are relative to papers we have ingested — papers, authors, and institutions outside the pilot are not represented. Methodology.

Top 100%percentile

0Author DataRank

Indexed papers

0in pilot corpus

datarank_citation_only_1hop_v6· scope data_onlyMethodology

Papers

Driven by 13 papers. Top paper: “Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors”.

Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors

Molecular Therapy — Methods & Clinical Development(2020)10.1016/j.omtm.2020.11.015

N/A

0DataRank · unranked

49 citations

Marialuisa Alliegro, Margherita Dell’Anno, Edoardo Nusco, John M. Cullen, Stephanie Smith +12 more

Promoterless, Nuclease‐Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia

Hepatology(2020)10.1002/hep.31570

N/A

0DataRank · unranked

55 citations

Leah E. Venturoni, Jing Liao, Brandon T. Hubbard, Jessica L. Schneller, Victoria Hoffmann +9 more

Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease

Molecular Therapy(2020)10.1016/j.ymthe.2020.12.031

N/A

0DataRank · unranked

50 citations

Christopher A. Miller, Lauren Shea, Xuntian Jiang, Miguel A. Guzmán, Randy J. Chandler +8 more

The Platform Vector Gene Therapies Project: Increasing the Efficiency of Adeno-Associated Virus Gene Therapy Clinical Trial Startup

Human Gene Therapy(2020)10.1089/hum.2020.259

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0DataRank · unranked

33 citations

Elizabeth A. Ottinger, Deanna Portero, Richa Madan Lomash, Asaf Alimardanov, Pramod Terse +35 more

ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia

Molecular Therapy — Methods & Clinical Development(2021)10.1016/j.omtm.2021.06.015

N/A

0DataRank · unranked

37 citations

Alicia Michaud, Gina L. Rizzo, Christopher Roy, Sheldon S. Leung, Stephanie L. Elkins +11 more

Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann–Pick disease type C1 mice

Life Science Alliance(2021)10.26508/lsa.202101040

N/A

0DataRank · unranked

25 citations

Alana L. Gibson, Tansy Gu, Laura L. Baxter, Benjamin E. Deverman, Keith Beadle +10 more

In vivo genome editing at the albumin locus to treat methylmalonic acidemia

Molecular Therapy — Methods & Clinical Development(2021)10.1016/j.omtm.2021.11.004

N/A

0DataRank · unranked

19 citations

Ciaran M. Lee, Leah E. Venturoni, Randy J. Chandler, Ang Li, Sangho Myung +6 more

Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemia

Molecular Therapy — Methods & Clinical Development(2021)10.1016/j.omtm.2021.04.005

N/A

0DataRank · unranked

6 citations

PamelaSara E. Head, Leah E. Venturoni, Randy J. Chandler, Charles P. Venditti, Francis J. May

Growth advantage of corrected hepatocytes in a juvenile model of methylmalonic acidemia following liver directed adeno-associated viral mediated nuclease-free genome editing

Molecular Genetics and Metabolism(2022)10.1016/j.ymgme.2022.06.011

N/A

0DataRank · unranked

14 citations

Randy J. Chandler, Jing Liao, Victoria Hoffmann, Nikhil Ramesh, Susana Gordo +3 more

Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9

Molecular Therapy — Methods & Clinical Development(2022)10.1016/j.omtm.2022.09.001

N/A

0DataRank · unranked

14 citations

Giovanni Di Pasquale, Jennifer L. Sloan, Samantha McCoy, Brandon T. Hubbard, Tina M. Kilts +4 more

Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience

Human Gene Therapy(2023)10.1089/hum.2022.232

N/A

0DataRank · unranked

10 citations

Oleg A. Shchelochkov, Randy J. Chandler, NIH PaVe-GT Team, Charles P. Venditti, Anne Pariser +38 more

Systemic gene therapy using an AAV44.9 vector rescues a neonatal lethal mouse model of propionic acidemia

Molecular Therapy — Methods & Clinical Development(2023)10.1016/j.omtm.2023.06.008

N/A

0DataRank · unranked

10 citations

Giovanni Di Pasquale, Eun-Young Choi, David K. Chang, Stephanie Smith, Jennifer L. Sloan +5 more

Systemic Treatment for Postnatal, Juvenile, and Runted Adult Mice by Retrobulbar Sinus Injection

Journal of Visualized Experiments(2024)10.3791/66809

N/A

0DataRank · unranked

0 citations

Randy J. Chandler, Darwin Romero