Kirsten E Coleman,
AST DIR, Research Admin
Department:
MD-POWELL GENE THERAPY CENTER
Business Phone:
(352) 273-6661
Business Email:
kee2000@ufl.edu
About Kirsten E Coleman
Kirsten Coleman is the Associate Director of the University of Florida Powell Gene Therapy Center Toxicology Core. She obtained both her B.S. and M.B.A. degrees from the University of Florida. After completing her undergraduate honors thesis in Dr. Terence Flotte’s laboratory in 2004, she joined the Toxicology Core to participate in the first-in-human trial of recombinant adeno-associated virus (rAAV) 2 for the treatment of alpha-1 antitrypsin deficiency. Since then, she has participated in bench to bedside research for multiple disorders utilizing the AAV gene therapy platform. Kirsten’s current responsibilities include designing and coordinating numerous aspects of preclinical and clinical testing, as well as overseeing that the Toxicology Core adheres to FDA Good Laboratory Practice (GLP) 21 CFR Part 58 regulations. Her research focuses on the testing of cell and gene therapy vectors to determine their biodistribution, immunogenicity, pathology and efficacy. Along with the team at UF, she has successfully developed FDA-approved investigational new drug applications for the treatment of a wide range of musculoskeletal, metabolic, ocular and neurological diseases.
Teaching Profile
Courses Taught
2020-2022
GMS6252 Molecular Therapy II – Disease Targets and Applications
Research Profile
Areas of Interest
- Adeno-Associated Viral Gene Therapy
- Gene therapy
- Gene therapy for cardiovascular disease
- Neurodegenerative diseases
- Pediatrics
- Rare neuromuscular disorders
Publications
Academic Articles
2024
RNA aggregates harness the danger response for potent cancer immunotherapy.
Cell.
187(10):2521-2535.e21
[DOI] 10.1016/j.cell.2024.04.003.
[PMID] 38697107.
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
A clinically viable approach to restoring visual function using optogenetic gene therapy.
Molecular therapy. Methods & clinical development.
29:406-417
[DOI] 10.1016/j.omtm.2023.05.005.
[PMID] 37251979.
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.
2022
Age-stratified adeno-associated virus serotype 3 neutralizing and total antibody prevalence in hemophilia A patients from India.
Journal of medical virology.
94(9):4542-4547
[DOI] 10.1002/jmv.27859.
[PMID] 35577570.
2021
A comprehensive study of a 29-capsid AAV library in a non-human primate central nervous system.
Molecular therapy : the journal of the American Society of Gene Therapy.
29(9):2806-2820
[DOI] 10.1016/j.ymthe.2021.07.010.
[PMID] 34298128.
2021
Prevalence of Adeno-Associated Virus 3 Capsid Binding and Neutralizing Antibodies in Healthy and Hemophilia B Individuals from India.
Human gene therapy.
32(9-10):451-457
[DOI] 10.1089/hum.2020.258.
[PMID] 33207962.
2021
Voluntary wheel running complements microdystrophin gene therapy to improve muscle function in mdx mice.
Molecular therapy. Methods & clinical development.
21:144-160
[DOI] 10.1016/j.omtm.2021.02.024.
[PMID] 33850950.
2019
Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study.
Molecular therapy. Methods & clinical development.
13:233-242
[DOI] 10.1016/j.omtm.2019.01.013.
[PMID] 30828586.
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
Long-term safety and efficacy of AAV gene therapy in the canine model of glycogen storage disease type Ia.
.
41(6):977-984
2018
Long-term safety and efficacy of AAV gene therapy in the canine model of glycogen storage disease type Ia.
Journal of inherited metabolic disease.
41(6):977-984
[DOI] 10.1007/s10545-018-0199-7.
[PMID] 29802554.
2018
Selective Neuronal Uptake and Distribution of AAVrh8, AAV9, and AAVrh10 in Sheep After Intra-Striatal Administration.
Journal of Huntington's disease.
7(4):309-319
[DOI] 10.3233/JHD-180302.
[PMID] 30320596.
2018
Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice.
Human gene therapy. Clinical development.
29(4):188-197
[DOI] 10.1089/humc.2018.168.
[PMID] 30280954.
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
In vitro evaluation of mitochondrial dysfunction and treatment with adeno-associated virus vector in fibroblasts from Doberman Pinschers with dilated cardiomyopathy and a pyruvate dehydrogenase kinase 4 mutation.
American journal of veterinary research.
77(2):156-61
[DOI] 10.2460/ajvr.77.2.156.
[PMID] 27027709.
2016
Reduced retinal transduction and enhanced transgene-directed immunogenicity with intravitreal delivery of rAAV following posterior vitrectomy in dogs.
Gene therapy.
23(6):548-56
[DOI] 10.1038/gt.2016.31.
[PMID] 27052802.
2016
Transfer of Therapeutic Genes into Fetal Rhesus Monkeys Using Recombinant Adeno-Associated Type I Viral Vectors.
Human gene therapy. Clinical development.
27(4):152-159
[PMID] 27855487.
2016
Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial.
Human genetics.
135(3):327-43
[DOI] 10.1007/s00439-016-1637-y.
[PMID] 26825853.
2015
Safety and Biodistribution Evaluation of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin, in RS1-Deficient Mice.
Human gene therapy. Clinical development.
26(3):177-84
[DOI] 10.1089/humc.2015.077.
[PMID] 26390091.
2015
Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-Linked Retinitis Pigmentosa.
Human gene therapy.
26(9):593-602
[DOI] 10.1089/hum.2015.035.
[PMID] 26076799.
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
LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile.
Investigative ophthalmology & visual science.
55(12):7739-53
[DOI] 10.1167/iovs.14-15388.
[PMID] 25342621.
2014
Reprogramming adipose tissue-derived mesenchymal stem cells into pluripotent stem cells by a mutant adeno-associated viral vector.
Human gene therapy methods.
25(1):72-82
[DOI] 10.1089/hgtb.2013.011.
[PMID] 24191859.
2013
Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa.
Human gene therapy. Clinical development.
24(1):23-8
[DOI] 10.1089/humc.2013.037.
[PMID] 23692380.
2013
Preclinical Safety Studies for Aav2-Mertk Gene Therapy Vector for Retinitis Pigmentosa
Molecular Therapy.
21
2013
Preclinical toxicology and biodistribution studies of recombinant adeno-associated virus 1 human acid α-glucosidase.
Human gene therapy. Clinical development.
24(3):127-33
[DOI] 10.1089/humc.2013.147.
[PMID] 24021025.
2013
Stem-cell therapy for dilated cardiomyopathy: a pilot study evaluating retrograde coronary venous delivery.
The Journal of small animal practice.
54(7):361-6
[DOI] 10.1111/jsap.12098.
[PMID] 23731226.
2012
Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.
Archives of ophthalmology (Chicago, Ill. : 1960).
130(1):9-24
[DOI] 10.1001/archophthalmol.2011.298.
[PMID] 21911650.
2012
Identification of adeno-associated viral vectors suitable for intestinal gene delivery and modulation of experimental colitis.
American journal of physiology. Gastrointestinal and liver physiology.
302(3):G296-308
[DOI] 10.1152/ajpgi.00562.2010.
[PMID] 22114116.
2012
Long-term correction of very long-chain acyl-coA dehydrogenase deficiency in mice using AAV9 gene therapy.
Molecular therapy : the journal of the American Society of Gene Therapy.
20(6):1131-8
[DOI] 10.1038/mt.2012.39.
[PMID] 22395529.
2012
Sex-Dependent Expression of Very Long Chain Acyl-Coa Dehydrogenase (Vlcad) Transgene and the Role of the Liver in Biochemical and Phenotypic Correction of Vlcad Deficiency
Molecular Therapy.
20
2012
The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina.
Human gene therapy.
23(10):1101-15
[DOI] 10.1089/hum.2012.125.
[PMID] 22845794.
2012
Transduction and Expression in Brain After Systemic Delivery of Single Stranded Aav9 in Adult Mice
Molecular Therapy.
20
2011
Clinical Manifestations of Heterozygous Expression of a Mutation That Causes Glycogen Storage Disease Type Ia in Dogs
Journal of Veterinary Internal Medicine.
25:731-732
2011
Glycogen storage disease type Ia in canines: a model for human metabolic and genetic liver disease.
Journal of biomedicine & biotechnology.
2011
[DOI] 10.1155/2011/646257.
[PMID] 21318173.
2011
Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse.
Investigative ophthalmology & visual science.
52(10):7098-108
[DOI] 10.1167/iovs.11-7867.
[PMID] 21778276.
2011
Next Generation Treatment of Pompe Disease Using Systemic Gene Transfer With Aav9
Molecular Genetics and Metabolism.
102(2)
[DOI] 10.1016/j.ymgme.2010.11.062.
2009
Preclinical Toxicology and Biodistribution Studies of Recombinant Adeno-Associated Virus 1 (Ravv1) Human Acid Alpha-Flucosidase (Gaa) in Nzw Rabbits By Intrathoracic Direct Injection in the Diaphragm
Molecular Therapy.
17
2007
In vivo expression of human ATP:cob(I)alamin adenosyltransferase (ATR) using recombinant adeno-associated virus (rAAV) serotypes 2 and 8.
The journal of gene medicine.
9(6):462-9
[PMID] 17471589.
2006
Systemic correction of a fatty acid oxidation defect by intramuscular injection of a recombinant adeno-associated virus vector.
Human gene therapy.
17(1):71-80
[PMID] 16409126.
2005
Efficient Hepatic Delivery and Expression from a Recombinant Adeno-associated Virus 8 Pseudotyped alpha1-Antitrypsin Vector
Molecular Therapy.
12(5):867-875
Presentations
2020-05-11
A Practical Guide to Preclinical Study Design
International
/
Invited.
Workshop.
2024-05-07
How to Design Phase-Appropriate Successful IND-Enabling Tox Studies for an AAV Drug Product
International
/
Invited.
Workshop.
Baltimore, MD
Grants
Jan 2024
– Dec 2024
Task Order#1 for UF #AGR00019321- Rocket Pharmaceuticals
Role: Co-Investigator
Funding: ROCKET PHARMACEUTICALS
Jan 2024
ACTIVE
Novel Gene Therapy for Critical Limb Ischemia and Limb Salvage in Vascular Occlusive Disease
Role: Co-Investigator
Funding: UNIV OF MIAMI
via NATL INST OF HLTH NHLBI
Jun 2023
ACTIVE
Measurement of Frataxin mRNA in NHP and human biofluids
Role: Project Manager
Funding: FRIEDREICHS ATAXIA RESEARCH ALLIANCE
Mar 2023
– Aug 2023
Task Order 17 – Biodistribution of the hFXN2 in mus musculus
Role: Other
Funding: SOLID BIOSCIENCES INC
Sep 2022
– Mar 2024
Preclinical sub-chronic toxicology and biodistribution study of adeno-associated virus (scAAV9-CBh-hAGAopt) mediated gene therapy for Aspartylglucosaminuria Disease in Sprague Dawley rats
Role: Other
Funding: RARE TRAIT HOPE FUND
Aug 2022
– Jul 2023
Cincinnati Zoo Feline and Canine Shedding 2022 Non-GLP
Role: Other
Funding: MASS GENERAL BRIGHAM
May 2021
ACTIVE
Immunomodulation Approaches to Improve Safety And Efficacy of Gene Therapy Treatment in Friedreichs Ataxia
Role: Co-Investigator
Funding: NATL INST OF HLTH NINDS
Mar 2021
– Feb 2022
Task Order #6: Assays for candidate AAV capsids
Role: Co-Investigator
Funding: ATSENA THERAPEUTICS
Dec 2020
– May 2021
Determination of anti-AAV9 IgG & anti-AAV9 IgM level in mouse serum samples
Role: Other
Funding: SOLID BIOSCIENCES INC
May 2020
– Dec 2020
Analysis of Cincinnati Zoo Feline Shedding
Role: Co-Investigator
Funding: MASS GENERAL BRIGHAM
Aug 2019
ACTIVE
Development of a Novel AAV Vector Capsid Optimized for OA Gene Therapy
Role: Co-Investigator
Funding: US ARMY MED RES ACQUISITION
Mar 2019
– Dec 2019
GLP Tissue-only Biodistribution Study in Nonhuman Primates
Role: Co-Investigator
Funding: APIC BIO
Dec 2018
– Mar 2019
Lacerta Task Order #5 – Preparation of DNA and RNA from NHP CNS tissues
Role: Principal Investigator
Funding: *LACERTA THERAPEUTICS
Oct 2018
– Dec 2018
Analysis of Cincinnati Zoo Feline Shedding
Role: Co-Investigator
Funding: MASS GENERAL BRIGHAM
Jul 2018
– Dec 2018
Limelight TO #1 – Optimizing Hydrodynamic Transduction Injection Procedure
Role: Co-Investigator
Funding: LIMELIGHT BIO
Nov 2017
– Feb 2019
AGTC synthetic promoter study
Role: Project Manager
Funding: APPLIED GENETICS TECH CORP
Nov 2017
– Jun 2018
Hex-003-004
Role: Co-Investigator
Funding: UNIV OF MASSACHUSETTS MEDICAL SCHOOL
Oct 2017
– Mar 2019
Toxicology Study of BS01 in Rd1 mice
Role: Co-Investigator
Funding: APPLIED GENETICS TECH CORP
Jul 2016
– Dec 2019
Clinical trial readiness for AAV-mediated gene therapy in Friedreichs Ataxia
Role: Project Manager
Funding: GOFAR
Education
Master of Business Administration
2009-2011
·
University of Florida
Bachelor of Science in Microbiology and Chemistry
2000-2004
·
University of Florida
Contact Details
Phones:
- Business:
- (352) 273-6661
Emails:
- Business:
- kee2000@ufl.edu
Addresses:
- Business Mailing:
-
PO Box 100292
GAINESVILLE FL 32610 - Business Street:
-
1200 NEWELL DR
GAINESVILLE FL 32610