World's Best Scientists 2026 revealed!

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Molecular Biology

D-Index
107
Citations
54643
World Ranking
407
National Ranking
234

Biology and Biochemistry

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118
Citations
60113
World Ranking
695
National Ranking
440

Research.com Recognitions

  • 2019 - Breakthrough Prize in Life Sciences for the development of an effective antisense oligonucleotide therapy for children with the neurodegenerative disease spinal muscular atrophy.

Overview

C. Frank Bennett is affiliated with Ionis Pharmaceuticals in the United States and has a significant body of work primarily in the fields of Biochemistry, Genetics and Molecular Biology, as well as Medicine. Their research encompasses key subfields including Molecular Biology, Genetics, Neurology, Cellular and Molecular Neuroscience, and Surgery.

The scientist's main topics of study focus on Neurogenetic and Muscular Disorders Research, RNA Research and Splicing, RNA modifications and cancer, Amyotrophic Lateral Sclerosis (ALS) Research, Genetic Neurodegenerative Diseases, Muscle Physiology and Disorders, and RNA regulation and disease.

Frank Bennett has contributed to several prominent research publications with papers appearing in high-impact journals. Notable recent papers include:

  • Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS, 2020, New England Journal of Medicine
  • Tau-targeting antisense oligonucleotide MAPTRx in mild Alzheimer's disease: a phase 1b, randomized, placebo-controlled trial, 2023, Nature Medicine
  • Mechanism of STMN2 cryptic splice-polyadenylation and its correction for TDP-43 proteinopathies, 2023, Science
  • Safety, tolerability and antiviral activity of the antisense oligonucleotide bepirovirsen in patients with chronic hepatitis B: a phase 2 randomized controlled trial, 2021, Nature Medicine
  • Cholesterol-functionalized DNA/RNA heteroduplexes cross the blood-brain barrier and knock down genes in the rodent CNS, 2021, Nature Biotechnology

Frequent co-authors in Bennett's work include Frank Rigo, Roger Lane, Karen Ling, Don W. Cleveland, and Holly Kordasiewicz. These collaborations indicate ongoing partnerships in advancing research related to antisense oligonucleotides and neurodegenerative diseases.

The scientist has published frequently in several notable venues, including bioRxiv (Cold Spring Harbor Laboratory), Nature Medicine, Human Molecular Genetics, Nature Communications, and Nucleic Acids Research. The diversity of these platforms reflects a broad impact across multiple areas within molecular genetics and neurological research.

Best Publications

  • miR-122 regulation of lipid metabolism revealed by in vivo antisense targeting.

    Christine Esau;Scott Davis;Susan F. Murray;Xing Xian Yu

  • The nuclear-retained noncoding RNA MALAT1 regulates alternative splicing by modulating SR splicing factor phosphorylation.

    Vidisha Tripathi;Jonathan D. Ellis;Zhen Shen;David Y. Song

  • Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

    Richard S. Finkel;Eugenio Mercuri;Basil T. Darras;Anne M. Connolly

  • RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

    C Frank Bennett;Eric E Swayze

  • Long pre-mRNA depletion and RNA missplicing contribute to neuronal vulnerability from loss of TDP-43

    Magdalini Polymenidou;Clotilde Lagier-Tourenne;Kasey R. Hutt;Stephanie C. Huelga

  • MicroRNA-143 regulates adipocyte differentiation.

    Christine Esau;Xiaolin Kang;Eigen Peralta;Elaine Hanson

  • Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

    Eugenio Mercuri;Basil T. Darras;Claudia A. Chiriboga;John W. Day

  • Antisense oligonucleotide modulation of multidrug resistance-associated protein

    Edgardo Baracchini;C. Frank Bennett;Nicholas M. Dean

  • RNA Toxicity from the ALS/FTD C9ORF72 Expansion Is Mitigated by Antisense Intervention

    Christopher J. Donnelly;Ping-Wu Zhang;Jacqueline T. Pham;Aaron R. Haeusler

  • Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

    Richard S Finkel;Claudia A Chiriboga;Jiri Vajsar;John W Day

  • Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides.

    Richard S. Geary;Daniel Norris;Rosie Yu;C. Frank Bennett

  • Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis.

    Holly B. Kordasiewicz;Lisa M. Stanek;Edward V. Wancewicz;Curt Mazur

  • Efficient Reduction of Target RNAs by Small Interfering RNA and RNase H-dependent Antisense Agents: A COMPARATIVE ANALYSIS *

    Timothy A. Vickers;Seongjoon Koo;C. Frank Bennett;Stanley T. Crooke

  • Divergent roles of ALS-linked proteins FUS/TLS and TDP-43 intersect in processing long pre-mRNAs

    Clotilde Lagier-Tourenne;Magdalini Polymenidou;Kasey R. Hutt;Anthony Q. Vu

  • Targeting RNA Foci in iPSC-Derived Motor Neurons from ALS Patients with a C9ORF72 Repeat Expansion

    Dhruv Sareen;Jacqueline G. O’Rourke;Pratap Meera;A. K. M. G. Muhammad

  • Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model

    Yimin Hua;Kentaro Sahashi;Frank Rigo;Gene Hung

  • RNA-Targeted Therapeutics.

    Stanley T. Crooke;Joseph L. Witztum;C. Frank Bennett;Brenda F. Baker

  • Regulating Gene Expression through RNA Nuclear Retention

    Kannanganattu V. Prasanth;Supriya G. Prasanth;Zhenyu Xuan;Stephen Hearn

  • Antisense Masking of an hnRNP A1/A2 Intronic Splicing Silencer Corrects SMN2 Splicing in Transgenic Mice

    Yimin Hua;Timothy A. Vickers;Hazeem L. Okunola;C. Frank Bennett

  • Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

    Yimin Hua;Kentaro Sahashi;Gene Hung;Frank Rigo

Frequent Co-Authors

Frank Rigo
Frank Rigo Ionis Pharmaceuticals (United States)
Nicholas M. Dean
Nicholas M. Dean Ionis Pharmaceuticals (United States)
Eric E. Swayze
Eric E. Swayze Ionis Pharmaceuticals (United States)
Adrian R. Krainer
Adrian R. Krainer Cold Spring Harbor Laboratory
Don W. Cleveland
Don W. Cleveland University of California, San Diego
David J. Ecker
David J. Ecker Ionis Pharmaceuticals (United States)
Brett P. Monia
Brett P. Monia Ionis Pharmaceuticals (United States)
David L. Spector
David L. Spector Cold Spring Harbor Laboratory
Punit P. Seth
Punit P. Seth Ionis Pharmaceuticals (United States)
Muthiah Manoharan
Muthiah Manoharan Alnylam Pharmaceuticals (United States)

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