Manuela Corti, Ph.D., P.T.
Associate Professor
Department:
MD-CHILD HEALTH RES INSTITUTE
Business Phone:
(352) 294-5779
Business Email:
m.corti@peds.ufl.edu
Teaching Profile
Courses Taught
2017-2020
GMS6001 Fundamentals of Biomedical Sciences I
2020-2022,2024
GMS6252 Molecular Therapy II – Disease Targets and Applications
2019-2021,2023-2024
GMS6847 Translational Research and Therapeutics: Bench, Bedside, Community, & Policy
2021
GMS5905 Special Topics in Biomedical Sciences
Research Profile
Open Researcher and Contributor ID (ORCID)
0000-0002-6391-4139
Publications
2024
B cell focused transient immune suppression protocol for efficient AAV readministration to the liver.
Molecular therapy. Methods & clinical development.
32(1)
[DOI] 10.1016/j.omtm.2024.101216.
[PMID] 38440160.
2024
Emerging therapies in hereditary ataxias.
Trends in molecular medicine.
[DOI] 10.1016/j.molmed.2024.07.008.
[PMID] 39153956.
2024
Longitudinal changes of swallowing safety and efficiency in infants with spinal muscular atrophy who received disease modifying therapies
Pediatric Pulmonology.
59(5):1364-1371
[DOI] 10.1002/ppul.26919.
2024
Perspectives of the Friedreich ataxia community on gene therapy clinical trials.
Molecular therapy. Methods & clinical development.
32(1)
[DOI] 10.1016/j.omtm.2023.101179.
[PMID] 38261944.
2024
The multifaceted roles of the brain glycogen
Journal of Neurochemistry.
168(5):728-743
[DOI] 10.1111/jnc.15926.
[PMID] 37554056.
2024
Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies.
The Journal of clinical investigation.
134(1)
[DOI] 10.1172/JCI173510.
[PMID] 37988172.
2023
Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation
Molecular Therapy – Methods & Clinical Development.
30:303-314
[DOI] 10.1016/j.omtm.2023.06.001.
[PMID] 37601414.
2023
Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy.
Science translational medicine.
15(677)
[DOI] 10.1126/scitranslmed.abo1815.
[PMID] 36599002.
2023
Evolving Horizons: Adenovirus Vectors’ Timeless Influence on Cancer, Gene Therapy and Vaccines.
Viruses.
15(12)
[DOI] 10.3390/v15122378.
[PMID] 38140619.
2023
On “Clinical and Research Readiness for Spinal Muscular Atrophy: The Time Is Now for Knowledge Translation.” Krosschell KJ, Young SD, Peterson I, et al. Phys Ther. 2022;102:pzac108. https://doi.org/10.1093/Ptj/pzac108.
Physical therapy.
103(3)
[DOI] 10.1093/ptj/pzad008.
[PMID] 37172132.
2022
A natural history study to track brain and spinal cord changes in individuals with Friedreich’s ataxia: TRACK-FA study protocol.
PloS one.
17(11)
[DOI] 10.1371/journal.pone.0269649.
[PMID] 36410013.
2022
Natural History of Friedreich Ataxia
Neurology.
99(14)
[DOI] 10.1212/wnl.0000000000200913.
2022
Secondary hemophagocytic lymphohistiocytosis following Zolgensma therapy: An evolving story on the innate response to systemic gene therapy.
Molecular therapy : the journal of the American Society of Gene Therapy.
30(12):3503-3504
[DOI] 10.1016/j.ymthe.2022.11.007.
[PMID] 36436512.
2021
Body Mass Index and Height in the Friedreich Ataxia Clinical Outcome Measures Study.
Neurology. Genetics.
7(6)
[DOI] 10.1212/NXG.0000000000000638.
[PMID] 34786480.
2021
Considerations for Systemic Use of Gene Therapy.
Molecular therapy : the journal of the American Society of Gene Therapy.
29(2):422-423
[DOI] 10.1016/j.ymthe.2021.01.016.
[PMID] 33485465.
2021
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Molecular therapy : the journal of the American Society of Gene Therapy.
29(2):464-488
[DOI] 10.1016/j.ymthe.2020.12.007.
[PMID] 33309881.
2021
Gauging Gait Disorders with a Method Inspired by Motor Control Theories: A Pilot Study in Friedreich’s Ataxia.
Sensors (Basel, Switzerland).
21(4)
[DOI] 10.3390/s21041144.
[PMID] 33562027.
2021
Resistance exercise training with protein supplementation improves skeletal muscle strength and improves quality of life in late adolescents and young adults with Barth syndrome: A pilot study.
JIMD reports.
62(1):74-84
[DOI] 10.1002/jmd2.12244.
[PMID] 34765401.
2021
Safety and Efficacy of Omaveloxolone in Friedreich Ataxia (MOXIe Study).
Annals of neurology.
89(2):212-225
[DOI] 10.1002/ana.25934.
[PMID] 33068037.
2021
Scratching the surface of RGD-directed AAV capsid engineering.
Molecular therapy : the journal of the American Society of Gene Therapy.
29(11):3099-3100
[DOI] 10.1016/j.ymthe.2021.10.020.
[PMID] 34699781.
2020
Advancements in AAV-mediated Gene Therapy for Pompe Disease.
Journal of neuromuscular diseases.
7(1):15-31
[DOI] 10.3233/JND-190426.
[PMID] 31796685.
2020
Management of Neuroinflammatory Responses to AAV-Mediated Gene Therapies for Neurodegenerative Diseases.
Brain sciences.
10(2)
[DOI] 10.3390/brainsci10020119.
[PMID] 32098339.
2020
Temporal but not spatial dysmetria relates to disease severity in FA.
Journal of neurophysiology.
123(2):718-725
[DOI] 10.1152/jn.00165.2019.
[PMID] 31693434.
2019
Pompe disease gene therapy: neural manifestations require consideration of CNS directed therapy.
Annals of translational medicine.
7(13)
[DOI] 10.21037/atm.2019.05.56.
[PMID] 31392202.
2018
Reduced Muscle Strength in Barth Syndrome May Be Improved by Resistance Exercise Training: A Pilot Study.
JIMD reports.
41:63-72
[DOI] 10.1007/8904_2018_102.
[PMID] 29654548.
2017
Inspiratory muscle conditioning exercise and diaphragm gene therapy in Pompe disease: Clinical evidence of respiratory plasticity.
Experimental neurology.
287(Pt 2):216-224
[DOI] 10.1016/j.expneurol.2016.07.013.
[PMID] 27453480.
2017
Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease.
Human gene therapy. Clinical development.
28(4):208-218
[DOI] 10.1089/humc.2017.146.
[PMID] 29160099.
2016
A majority rule approach for region-of-interest-guided streamline fiber tractography.
Brain imaging and behavior.
10(4):1137-1147
[PMID] 26572144.
2016
Altered activation of the diaphragm in late-onset Pompe disease.
Respiratory physiology & neurobiology.
222:11-5
[DOI] 10.1016/j.resp.2015.11.013.
[PMID] 26612101.
2016
Reply: Respiratory motor function in centronuclear myopathy.
Muscle & nerve.
53(4):660-1
[DOI] 10.1002/mus.24961.
[PMID] 26561983.
2016
Respiratory motor function in individuals with centronuclear myopathies.
Muscle & nerve.
53(2):214-21
[DOI] 10.1002/mus.24899.
[PMID] 26351754.
2016
Targeted approaches to induce immune tolerance for Pompe disease therapy.
Molecular therapy. Methods & clinical development.
3
[DOI] 10.1038/mtm.2015.53.
[PMID] 26858964.
2015
Altered activation of the tibialis anterior in individuals with Pompe disease: Implications for motor unit dysfunction.
Muscle & nerve.
51(6):877-83
[DOI] 10.1002/mus.24444.
[PMID] 25186912.
2015
Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning.
Human gene therapy. Clinical development.
26(3):185-93
[DOI] 10.1089/humc.2015.068.
[PMID] 26390092.
2014
B-Cell Depletion is Protective Against Anti-AAV Capsid Immune Response: A Human Subject Case Study.
Molecular therapy. Methods & clinical development.
1
[PMID] 25541616.
2014
Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAaV1-CMV-GAA) gene vector in patients with Pompe disease.
Human gene therapy. Clinical development.
25(3):134-63
[DOI] 10.1089/humc.2014.2514.
[PMID] 25238277.
2014
Selective Knockout of Acid Alpha-Glucosidase Gaa Gene Expression in the Medulla Alters Breathing
The FASEB's Journal.
28(1, S)
2013
Adeno-associated virus-mediated gene therapy for metabolic myopathy.
Human gene therapy.
24(11):928-36
[DOI] 10.1089/hum.2013.2514.
[PMID] 24164240.
2013
Magnetic Resonance Imaging (Mri) To Evaluate the Effect of Enzyme Replacement Therapy in Late Onset Pompe Disease (Lopd)
Neuromuscular Disorders.
23(9-10)
[DOI] 10.1016/j.nmd.2013.06.654.
2013
Magnetic Resonance Imaging and Spectroscopy To Evaluate Muscle Involvement in Pompe Disease
Molecular Genetics and Metabolism.
108(2)
[DOI] 10.1016/j.ymgme.2012.11.059.
2013
The respiratory neuromuscular system in Pompe disease.
Respiratory physiology & neurobiology.
189(2):241-9
[DOI] 10.1016/j.resp.2013.06.007.
[PMID] 23797185.
2012
Differential effects of power training versus functional task practice on compensation and restoration of arm function after stroke.
Neurorehabilitation and neural repair.
26(7):842-54
[DOI] 10.1177/1545968311433426.
[PMID] 22357633.
2012
Repetitive transcranial magnetic stimulation of motor cortex after stroke: a focused review.
American journal of physical medicine & rehabilitation.
91(3):254-70
[DOI] 10.1097/PHM.0b013e318228bf0c.
[PMID] 22042336.
Grants
Dec 2023
ACTIVE
Frataxin Potency assay in support of an AAV-mediated gene therapy treatment for Friedreichs ataxia
Role: Principal Investigator
Funding: FRIEDREICHS ATAXIA RESEARCH ALLIANCE
Oct 2023
– May 2024
Cardiac MRI outcomes in Friedreich's Ataxia
Role: Principal Investigator
Funding: REATA PHARMACEUTICALS
Mar 2023
– Aug 2023
Task Order 17 – Biodistribution of the hFXN2 in mus musculus
Role: Principal Investigator
Funding: SOLID BIOSCIENCES INC
Sep 2022
– Aug 2024
Functional electrical stimulation (FES) cycling training to improve motor and cardiac functions in patients with Friedreichs Ataxia: a feasibility and efficacy study
Role: Principal Investigator
Funding: FRIEDREICHS ATAXIA RESEARCH ALLIANCE
Sep 2021
– Aug 2023
Measuring cardiopulmonary responses to the 6MWT and peak effort exercise in DMD
Role: Co-Investigator
Funding: MUSCULAR DYSTROPHY ASSO
Jul 2021
– Jun 2022
Biodistribution and total anti-AAV antibody assays to support Friedreichs Ataxia preclinical studies
Role: Principal Investigator
Funding: *AAVANTIBIO
May 2021
– Jun 2021
CMS, COHBI – Powell Research for Rare Disease
Role: Faculty
Funding: FL DEPT OF HLTH
May 2021
ACTIVE
Immunomodulation Approaches to Improve Safety And Efficacy of Gene Therapy Treatment in Friedreichs Ataxia
Role: Principal Investigator
Funding: NATL INST OF HLTH NINDS
Feb 2021
– Jun 2022
Task Order 9 – Friedreich Ataxia Research Support
Role: Principal Investigator
Funding: *AAVANTIBIO
Nov 2020
ACTIVE
A natural history study to TRACK brain and spinal cord changes in individuals with Friedreich Ataxia
Role: Principal Investigator
Funding: FRIEDREICHS ATAXIA RESEARCH ALLIANCE
Jul 2020
– Jul 2022
A New Understanding of Muscle Atrophy in Neuromuscular Disease
Role: Co-Investigator
Funding: UF RESEARCH
Jun 2020
– May 2023
Immune response to AAV Vector Capsid and Assessment of Eligibility for AAV-mediated Gene Therapy for Duchenne Muscular Dystrophy
Role: Principal Investigator
Funding: DUCHENNE UK
May 2020
ACTIVE
Control of Breathing and Pompe Disease
Role: Co-Investigator
Funding: NATL INST OF HLTH NICHD
Dec 2019
– Dec 2022
Sarepta TO #1: Support of PGTC Cores
Role: Project Manager
Funding: SAREPTA THERAPEUTICS
Dec 2019
– Nov 2021
Task Order 1: Friedreich Ataxia Research Support
Role: Principal Investigator
Funding: *AAVANTIBIO
Sep 2019
– Sep 2021
Immune modulation approaches with MGD010 molecule to improve safety and efficacy of gene therapy strategies.
Role: Principal Investigator
Funding: PROVENTION BIO
Jul 2019
– Dec 2020
Sarepta Task Order #7 – Pompe Canine Colony Housing Program
Role: Co-Investigator
Funding: SAREPTA THERAPEUTICS
Mar 2019
– Apr 2020
AAV-mediated gene therapy in Pompe rodent models
Role: Principal Investigator
Funding: *LACERTA THERAPEUTICS
Jan 2019
– Dec 2020
Management of AAV preimmunity for the treatment of Duchenne Muscular Dystrophy: from preclinical to clinical application
Role: Principal Investigator
Funding: DUCHENNE RESEARCH FUND
Oct 2018
– Aug 2021
Evaluation of Re-administration of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV9-DES-hGAA) in Patients with Late-Onset Pompe Disease (LOPD).
Role: Principal Investigator
Funding: *LACERTA THERAPEUTICS
Apr 2018
– Dec 2018
Lacerta Task Order #3 – NHP TAb Screening
Role: Principal Investigator
Funding: *LACERTA THERAPEUTICS
Jan 2018
– Jan 2020
Lacerta TO #2 – Adeno-associated viral vector (AAV) optimization for central nervous system targeted therapy in Pompe disease
Role: Principal Investigator
Funding: *LACERTA THERAPEUTICS
Jun 2017
– Jun 2022
Cure FA Research Foundation
Role: Project Manager
Funding: UF FOUNDATION
May 2017
– Nov 2019
Process Development for AAV Manufacturing of AAV-CB-hFXN
Role: Project Manager
Funding: CURE FA FOUNDATION
Feb 2017
– Jan 2020
AAV-mediated Gene Correction in Friedreich's Ataxia
Role: Principal Investigator
Funding: MUSCULAR DYSTROPHY ASSO
Sep 2016
– Aug 2019
Clinical Outcome Measures of Efficacy in Treatment of Friedreichs Ataxia
Role: Principal Investigator
Funding: FRIEDREICHS ATAXIA RESEARCH ALLIANCE
Jul 2016
– Dec 2019
Clinical trial readiness for AAV-mediated gene therapy in Friedreichs Ataxia
Role: Principal Investigator
Funding: GOFAR
Nov 2015
– Nov 2017
EVALUATION OF THE EFFICACY OF RAAV9-CBA-HFXN & RAV6-CBA-HFXN AND RAV6-CBA-HFXN IN TRANSDUCING THE CNS IN RHESUS MACAQUES
Role: Principal Investigator
Funding: CYDAN DEVELOPMENT INC
Aug 2015
– Jun 2023
Diet and Exercise in pompe disease
Role: Principal Investigator
Funding: AMICUS THERAPEUTICS
May 2015
– Jun 2023
FRIEDREICH'S ATAXIA GENE THERAPY RESEARCH FUND
Role: Principal Investigator
Funding: UF FOUNDATION
Apr 2015
– Jun 2018
AAV9-Adult Study: Evaluation of Re-administration of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV9-DES-hGAA) in Patients with Late-Onset Pompe Disease (LOPD)
Role: Principal Investigator
Funding: AUDENTES THERAPEUTICS
Aug 2014
ACTIVE
CORTI 214 CLINICAL TRIAL OPERATING ACCOUNT
Role: Principal Investigator
Funding: UNIV OF FLORIDA
Contact Details
Phones:
- Business:
- (352) 294-5779
Emails:
- Business:
- m.corti@peds.ufl.edu
Addresses:
- Business Mailing:
-
2004 MOWRY RD
GAINESVILLE FL 32610 - Business Street:
-
2004 MOWRY RD 2225
GAINESVILLE FL 32610