UF Health Cancer Center

Analysis of effects of early vs late gene therapy in MPS IIIB mice


Dr. Coy Heldermon

Email
coy.heldermon@medicine.ufl.edu

Phone
(352) 273-7497

Faculty Department/Division
Hematology & Oncology

This project is primarily:
Translational

Research Project Description:
We will be injecting and analyzing the effects of AAV and stem cell based gene therapy at early and late time points for distribution and effect on disease phenotype.

Does this project have an international component or travel?
No

Evaluation of late injection of an AAV gene therapy on brain and gut manifestations of Sanfilippo Syndrome

Dr. Coy Heldermon

Phone
(352) 273-7497

Faculty Department/Division
Hematology & Oncology

This project is primarily:
Translational

Research Project Description:
Stem cell sources will be transduced with a gene therapy vector and injected into mice with the disease. Effects on behavior,biochemistry and histology will be compared.

Does this project have an international component or travel?
No

Stem cell migration in CNS

Dr. Coy Heldermon

Email
coy.heldermon@medicine.ufl.edu

Phone
(352) 273-7497

Faculty Department/Division
Hematology & Oncology

This project is primarily:
Translational

Research Project Description:
neural and mesenchymal stem cells will be injected into mice via several methods and patterns of spread will be assessed using acombination of immuno-histological methods.

Does this project have an international component or travel?
No

IRAK-4 inhibition in combination with immunotherapy and targeted therapy for metastatic melanoma

Dr. Bently Doonan

Email
bently.doonan@medicine.ufl.edu

Phone
(352) 273-9894

Faculty Department/Division
Hematology & Oncology

This project is primarily:
Translational

Research Project Description:
Melanoma is a growing public health concern and poised to become the second most common malignancy by 2040. Immunotherapy and targeted therapy have revolutionized the treatment of metastatic melanoma, yet roughly 50% of patients ultimately succumb to their disease. Our group is studying a novel pathway in melanoma the MyD88/IRAK-4 inflammasome pathway as a potential target in combination with either immunotherapy or targeted therapy with specific focus on targeting melanoma brain metastases as these represent the major cause of melanoma associated mortality. Students involved in this study will perform both cell culture based experiments and mouse experiments pairing an oral IRAK-4 inhibitor with either immunotherapy (PD1 inhibition, LAG3 inhibition, CTLA4 inhibition) or targeted therapy (BRAF inhibition/MEK inhibition). The combination of this therapy with PD1 inhibition is planned for a first in human clinical trial set to open in February 2023 here at UF so there would also be potential for involvement with clinical subjects.

Does this project have an international component or travel?
No

Feasibility of developing a 3-dimensional “tumoroid” model of cancer


Dr. John Ligon

Email
john.ligon@ufl.edu

Phone
(352) 294-5559

Faculty Department/Division
UF Health Cancer Center

This project is primarily:
Translational

Research Project Description:
This project will assess the feasibility of developing a 3-dimensional “tumoroid” model of cancer. We will develop models from 3 sources: 1) mice injected with osteosarcoma K7M2 cells; 2) pet-dogs being treated at the College of Veterinary Medicine with osteosarcoma; 3) human patients being treated at Shands with osteosarcoma. We will compare these models to real tumors to determine how closely our models approximate the actual tumor immune microenvironement of human osteosarcoma. These models will enable us to then test novel treatments and determine: 1) if the treatments may be able to work in osteosarcoma; and 2) the mechanism by which these novel therapies work in terms of how individual cells interact with each other.

The medical student would be responsible for establishing these models through cell culture and maintaining the cell culture through replenishing the cell culture media. Based on the productivity over the summer, there would be potential to present the results as a poster and to potentially be included as an author on all related manuscripts.

This research is funded by a Hyundai Hope on Wheels grant which has been awarded to Dr. Ligon

Does this project have an international component or travel?
No

If your project has an international component please give details (where, when, data collection involved, etc.):
N/A