Manuela Corti

Manuela Corti, MD

Research Assistant Professor

Department: MD-CHILD HEALTH RES INSTITUTE
Business Phone: (352) 294-5779
Business Email: m.corti@peds.ufl.edu

Publications

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
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
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
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

Sep 2021 ACTIVE
Measuring cardiopulmonary responses to the 6MWT and peak effort exercise in DMD
Role: Co-Investigator
Funding: MUSCULAR DYSTROPHY ASSO
Jul 2021 ACTIVE
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 ACTIVE
Task Order 9 – Friedreich Ataxia Research Support
Role: Principal Investigator
Funding: AAVANTIBIO
Jul 2020 ACTIVE
A New Understanding of Muscle Atrophy in Neuromuscular Disease
Role: Co-Investigator
Funding: UF RESEARCH
Jun 2020 ACTIVE
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 ACTIVE
Task Order 1: Friedreich Ataxia Research Support
Role: Principal Investigator
Funding: AAVANTIBIO
Dec 2019 – Dec 2020
Sarepta TO #1: Support of PGTC Cores
Role: Project Manager
Funding: SAREPTA THERAPEUTICS
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
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 ACTIVE
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: FRIEDRICH'S 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 – Aug 2023
Diet and Exercise in pompe disease
Role: Principal Investigator
Funding: AMICUS THERAPEUTICS
May 2015 ACTIVE
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

Patents

Published April 2015
Methods of Permitting a Subject to Receive Multiple Doses of Recombinant Adeno-Associated Vrus
#US2017/0049887

Teaching Profile

Courses Taught
2017-2020
GMS6001 Fundamentals of Biomedical Sciences I
2020-2021
GMS6252 Molecular Therapy II ? Disease Targets and Applications
2019-2021
GMS6847 Translational Research and Therapeutics: Bench, Bedside, Community, & Policy
2021
GMS5905 Special Topics in Biomedical Sciences

Contact Details

Phones:
Business:
(352) 294-5779
Emails: