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
Publications
Academic Articles
2025
Cardiopulmonary exercise testing as an integrative approach to explore physiological limitations in Duchenne muscular dystrophy.
Journal of neuromuscular diseases.
[DOI] 10.1177/22143602251319170.
[PMID] 40033993.
2025
Correction: A natural history study to track brain and spinal cord changes in individuals with Friedreich’s ataxia: TRACK-FA study protocol.
PloS one.
20(3)
[DOI] 10.1371/journal.pone.0320683.
[PMID] 40100780.
2025
Current Clinical Applications of AAV-mediated Gene Therapy.
Molecular therapy : the journal of the American Society of Gene Therapy.
[DOI] 10.1016/j.ymthe.2025.04.045.
[PMID] 40329530.
2025
Emerging therapies in hereditary ataxias.
Trends in molecular medicine.
31(2):181-194
[DOI] 10.1016/j.molmed.2024.07.008.
[PMID] 39153956.
2025
Neuroimaging Biomarkers for Friedreich Ataxia: A Cross-Sectional Analysis of the TRACK-FA Study.
Annals of neurology.
[DOI] 10.1002/ana.27237.
[PMID] 40119735.
2025
Quantification and comparison of anti-AAV9 and anti-AAVrh74 antibodies in plasma and human milk: Implications for AAV-based gene therapy candidacy
Journal of Neuromuscular Diseases.
[DOI] 10.1177/22143602251324857.
[PMID] 40207793.
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
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
Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease.
bioRxiv : the preprint server for biology.
[DOI] 10.1101/2024.12.10.627800.
[PMID] 39763722.
2024
Neurological glycogen storage diseases and emerging therapeutics.
Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics.
21(5)
[DOI] 10.1016/j.neurot.2024.e00446.
[PMID] 39277505.
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: Heterogeneity of Neurologic Progression and Consequences for Clinical Trial Design.
Neurology.
99(14):e1499-e1510
[DOI] 10.1212/WNL.0000000000200913.
[PMID] 35817567.
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
ACTIVE
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