Laura P Ranum

Laura P Ranum

PROF

Department: Molecular Genetics & Microbiology
Business Phone: (352) 294-5209
Business Email: ranum@ufl.edu

Publications

2020
Loss of MBNL1 induces RNA misprocessing in the thymus and peripheral blood.
Nature communications. 11(1) [DOI] 10.1038/s41467-020-15962-x. [PMID] 32332745.
2020
Survival and Motor Phenotypes in FVB C9-500 ALS/FTD BAC Transgenic Mice Reproduced by Multiple Labs
Neuron. 108(4):784-796.e3 [DOI] 10.1016/j.neuron.2020.09.009. [PMID] 33022226.
2020
Metformin inhibits RAN translation through PKR pathway and mitigates disease inC9orf72ALS/FTD mice
Proceedings of the National Academy of Sciences. 117(31):18591-18599 [DOI] 10.1073/pnas.2005748117.
2019
Antibody Therapy Targeting RAN Proteins Rescues C9 ALS/FTD Phenotypes in C9orf72 Mouse Model
Neuron. 105(4):645-662.e11 [DOI] 10.1016/j.neuron.2019.11.007. [PMID] 31831332.
2019
Repeat-Associated Non-ATG Translation: Molecular Mechanisms and Contribution to Neurological Disease
Annual review of neuroscience (Online). 42:227-247 [DOI] 10.1146/annurev-neuro-070918-050405. [PMID] 30909783.
2019
Repeat-associated non-AUG (RAN) translation: insights from pathology
Laboratory Investigation. 99(7):929-942 [DOI] 10.1038/s41374-019-0241-x. [PMID] 30918326.
2018
Repeat-Associated Non-ATG Translation in Neurological Diseases.
Cold Spring Harbor perspectives in biology. 10(12) [DOI] 10.1101/cshperspect.a033019. [PMID] 29891563.
2018
SCA8 RAN polySer protein preferentially accumulates in white matter regions and is regulated by eIF3F.
The EMBO journal. 37(19) [DOI] 10.15252/embj.201899023. [PMID] 30206144.
2017
RAN Translation Regulated by Muscleblind Proteins in Myotonic Dystrophy Type 2
Neuron. 95(6):1292-1305.e5 [DOI] 10.1016/j.neuron.2017.08.039.
2015
Genome modification leads to phenotype reversal in human myotonic dystrophy type 1 induced pluripotent stem cell-derived neural stem cells.
Stem cells (Dayton, Ohio). 33(6):1829-38 [DOI] 10.1002/stem.1970. [PMID] 25702800.
2015
RAN Translation in Huntington Disease
Neuron. 88(4):667-677 [DOI] 10.1016/j.neuron.2015.10.038.
2014
Hippocampal sclerosis dementia with the C9ORF72 hexanucleotide repeat expansion.
Neurobiology of aging. 35(10):2419.e17-21 [DOI] 10.1016/j.neurobiolaging.2014.04.009. [PMID] 24819148.
2014
Repeat associated non-ATG (RAN) translation: new starts in microsatellite expansion disorders.
Current opinion in genetics & development. 26:6-15 [DOI] 10.1016/j.gde.2014.03.002. [PMID] 24852074.
2014
Mutant β-III spectrin causes mGluR1α mislocalization and functional deficits in a mouse model of spinocerebellar ataxia type 5.
The Journal of neuroscience : the official journal of the Society for Neuroscience. 34(30):9891-904 [DOI] 10.1523/JNEUROSCI.0876-14.2014. [PMID] 25057192.
2013
Compound Loss of Muscleblind-Like Function in Myotonic Dystrophy
Embo Molecular Medicine. 5:1887-1900
2013
RAN proteins and RNA foci from antisense transcripts in C9ORF72 ALS and frontotemporal dementia.
Proceedings of the National Academy of Sciences of the United States of America. 110(51):E4968-77 [DOI] 10.1073/pnas.1315438110. [PMID] 24248382.
2013
Repeat-Associated Non-Atg (Ran) Translation in Neurological Disease
Human Molecular Genetics. 22(R1):R45-R51 [DOI] 10.1093/hmg/ddt371. [PMID] 23918658.
2012
C9Orf72 Repeat Expansion in Amyotrophic Lateral Sclerosis in the Kii Peninsula of Japan
Archives of Neurology. 69(9):1154-1158 [DOI] 10.1001/archneurol.2012.1219. [PMID] 22637429.
2012
Ggcctg Repeats Put a Hex On Purkinje Cells and Motor Neurons in Sca36
Neurology. 79(4):302-303 [DOI] 10.1212/WNL.0b013e31826043d9. [PMID] 22744663.
2012
Mouse Model of Muscleblind-Like 1 Overexpression: Skeletal Muscle Effects and Therapeutic Promise
Human Molecular Genetics. 21(21):4645-4654 [DOI] 10.1093/hmg/dds306. [PMID] 22846424.
2011
Frequency of Kcnc3 Dna Variants as Causes of Spinocerebellar Ataxia 13 (Sca13)
PLoS One. 6(3) [DOI] 10.1371/journal.pone.0017811. [PMID] 21479265.
2011
Non-Atg-Initiated Translation Directed By Microsatellite Expansions
Proceedings of the National Academy of Sciences. 108(1):260-265 [DOI] 10.1073/pnas.1013343108. [PMID] 21173221.
2009
Rna Gain-Of-Function in Spinocerebellar Ataxia Type 8
Plos Genetics. 5(8) [DOI] 10.1371/journal.pgen.1000600. [PMID] 19680539.

Grants

Sep 2020 ACTIVE
Identifying and understanding the role of repeat RNAs and RAN proteins in Alzheimer's disease
Role: Mentor
Funding: NATL INST OF HLTH NIA
Apr 2020 ACTIVE
An endobody vaccine against a combination of RAN proteins as a therapeutic approach in a C9 ALS BAC mouse model
Role: Principal Investigator
Funding: JOHNS HOPKINS UNIV
Jan 2020 ACTIVE
Provenance Initiatives Fund at the Center for NeuroGenetics
Role: Principal Investigator
Funding: UF FOU
Nov 2019 ACTIVE
C9ORF72 ALS: Endobody vaccine for the treatment of C9 ALS/FTD
Role: Mentor
Funding: AMYOTROPHIC LATERAL SCLEROSIS ASSOC
Sep 2019 ACTIVE
Therapeutic Potential of Metformin, an FDA-approved drug, and PKR Targeting for C9orf72 ALS
Role: Principal Investigator
Funding: US ARMY MED RES AND MATERIAL COM
Dec 2018 ACTIVE
Presymptomatic biomarkers of C9-ALS/FTD
Role: Principal Investigator
Funding: AMYOTROPHIC LATERAL SCLEROSIS ASSOC
Sep 2018 – Apr 2020
Assessment of endobody vaccine for effectiveness and ability to generate C9 RAN antibodies
Role: Principal Investigator
Funding: UNITED NEUROSCIENCE LIMITED
Aug 2018 ACTIVE
The contribution of RAN proteins to C9ORF72 ALS/FTD
Role: Principal Investigator
Funding: MUSCULAR DYSTROPHY ASSO
Aug 2018 ACTIVE
C9ORF72 ALS: Proof of biology for antibody treatment in C9-ALS mouse model
Role: Principal Investigator
Funding: BIOGEN MA INC
Feb 2018 – Feb 2020
Therapeutic potential of CRISPR-Cas9 in C9ORF72-repeat deletion strategy in C9ORF72 BAC transgenic mice
Role: Principal Investigator
Funding: CRISPR THERAPEUTICS AG
Jan 2018 – Dec 2019
Molecular characterization of RNA and RAN protein effects in DM2
Role: Mentor
Funding: WYCK FOUNDATION
Jul 2017 ACTIVE
Neurodegenerative disease research
Role: Principal Investigator
Funding: UF FOU
Jul 2017 ACTIVE
University of Florida/National Ataxia Foundation Brain Donation Program
Role: Principal Investigator
Funding: NATL ATAXIA FOUNDATION
May 2017 ACTIVE
Therapeutic potential of CRISPR-Cas9 in C9ORF72 ALS/FTD-repeat deletion and transcriptional repression strategies in humanized C9ORF72 BAC transgenic mice
Role: Principal Investigator
Funding: TARGET ALS FOUNDATION via CRISPR THERAPEUTICS AG
Jan 2017 – Jun 2020
Developing human-derived antibodies to target dipeptide-repeat protein toxicity in C9orf72 disease
Role: Principal Investigator
Funding: TARGET ALS FOUNDATION
Jan 2017 – Jun 2020
Nuclear Export Inhibitor KPT-350 for C9orf72 and Sporadic ALS
Role: Principal Investigator
Funding: TARGET ALS FOUNDATION
Aug 2016 – Jul 2018
Molecular characterization & antibody therapy in a novel C9orf72 BAC mouse model
Role: Principal Investigator
Funding: MUSCULAR DYSTROPHY ASSO
Aug 2016 – Oct 2017
Developing a high-throughput screening assay for identification of small molecules that selectively target transcription of G4C2/G2C4 repeat expansions in the gene C9ORF72 in ALS
Role:
Funding: AMYOTROPHIC LATERAL SCLEROSIS ASSOC
Aug 2016 ACTIVE
Molecular Characterization of ALS/FTD in a novel C9orf72 BAC mouse model.
Role: Principal Investigator
Funding: NATL INST OF HLTH NINDS
Mar 2016 – Mar 2018
The Therapeutic Efficacy of Neurimmune?s Human Derived R
Role: Principal Investigator
Funding: NEURIMMUNE AG
Mar 2016 ACTIVE
neurodegenerative disease research
Role: Principal Investigator
Funding: UF FOU
Jan 2016 ACTIVE
Molecular genetic characterization of SCA8
Role: Principal Investigator
Funding: UF FOU
Jan 2016 ACTIVE
Molecular Genetics of the G4C2 expansion mutation in ALS/FTD
Role: Principal Investigator
Funding: UF FOU
Jan 2016 – Dec 2016
ASO targeting of bidirectional transcripts and RAN
Role: Principal Investigator
Funding: NATL ATAXIA FOUNDATION
Dec 2015 ACTIVE
Brainstorming microsatellite expansion diseases symposium
Role: Principal Investigator
Funding: UF FOU
Aug 2015 ACTIVE
Molecular Genetic Characterization of SCA8
Role: Principal Investigator
Funding: NATL INST OF HLTH NINDS
Jan 2015 – Jan 2018
MBI Research Program MGOLD1
Role: Principal Investigator
Funding: UF FOU
Sep 2013 – Jun 2019
Myotonic Dystrophy: Molecular Pathophysiology and CNS Effects
Role: Principal Investigator
Funding: NATL INST OF HLTH NINDS
Jul 2013 ACTIVE
Neurogenetics Fund
Role: Principal Investigator
Funding: UF FOU
Jul 2013 – Jun 2019
Matching Funds for PPG Project Titled: "Myotonic Dystrop
Role: Principal Investigator
Funding: FL CLINICAL PRACTICE ASSO
May 2013 – Apr 2016
Repeat asscoiated non-ATG (RAN) Translation in
Role: Principal Investigator
Funding: COLUMBIA UNIVERSITY

Patents

Published March 2014
Use and Treatment of Di-Amino Acid Repeat-Containing Proteins Associated with ALS
#US2016-0025747-A1
Published May 2016
Methods for Diagnosing Huntington's Disease
#US18/0292416

Teaching Profile

Courses Taught
2018
GMS7980 Research for Doctoral Dissertation
2018
GMS7979 Advanced Research
2016-2017
GMS6750 Molecular Pathobiology of Neural Disease

Contact Details

Phones:
Business:
(352) 294-5209
Emails:
Business:
ranum@ufl.edu