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Virginia Kimonis

Professor, Neurology · University of California

Credentials: · · · · ·

History

University of California
Present
University of California
Present

Pediatrics
Massachusetts General Hospital
1976–1979
UNIV OF SOUTHAMPTON, FAC OF MED, SOUTHAMPTON, UK
1972–1976

Research

Mutations in prion-like domains in hnRNPA2B1 and hnRNPA1 cause multisystem proteinopathy and ALS
Nature · 2013 · 1,248 citations
Inclusion body myopathy associated with Paget disease of bone and frontotemporal dementia is caused by mutant valosin-containing protein
Nature Genetics · 2004 · Last of 9 authors · 1,118 citations
Clinical manifestations in 105 persons with nevoid basal cell carcinoma syndrome
Review · American Journal of Medical Genetics · 1997 · First of 8 authors · 625 citations

JG
June-Anne Gold
29 papers
Merlin Butler
25 papers
TM
Tahseen Mozaffar
19 papers
Daniel Driscoll
18 papers
JM
Jennifer Miller
15 papers
Conrad Weihl
10 papers
Manaswitha Khare
10 papers · 7 as a resident
AP
Alan Pestronk
7 papers
John Weiss
7 papers
LT
Lac Ta
7 papers · 2 as a resident
LG
Liliane Gibbs
7 papers
ED
Eric Dec
6 papers · 2 as a resident

Grants

GYS1 Oligonucleotide Treatment as a Substrate Inhibitor for Pompe Disease
National Institute of Child Health and Human Development · R21
Contact PI · $431.8K · University of California-Irvine
2026–2028
Correction of VCP Disease Pathology with Antisense Oligonucleotides
National Institute on Aging · R21
Contact PI · $431.8K · University of California-Irvine
2026–2028
Epigenetic Silencing of VCP via an ASO-Conjugate Based Induced Proximity System for the Treatment of FTD-ALS in VCP Disease
National Institute of Neurological Disorders and Stroke · R41
PI · $306.9K · Junipero Therapeutics, Inc.
2026–2027
Mechanistic and Translational Investigations of HSPB8-Associated Dominant Rimmed Vacuolar Myopathy
National Institute of Arthritis and Musculoskeletal and Skin Diseases · R21
Contact PI · $368.8K · University of California-Irvine
2024–2026

Clinical trials

Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.
Principal investigator · Completed
University of California, Irvine
2015–2019
Clinical Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome
Principal investigator · Completed · Phase 1, Phase 2
Essentialis, Inc.
2014–2015
Characterization of Familial Myopathy and Paget Disease of Bone
Principal investigator · Recruiting
University of California, Irvine
2010–2030
Characterization of Inclusion Body Myopathy Associated With Paget's Disease of Bone and Frontotemporal Dementia (IBMPFD)
Principal investigator · Recruiting
University of California, Irvine
2007–2030