Exosome:Adeno-associated virus formulations for enhanced gene transfer

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Our project aims to enhance the use of adeno-associated viruses (AAVs) as gene therapy vectors, the student will learn to make these viruses and modify them to improve their therapeutic capacity, particularly in central nervous system disorders.

Project description

The goal of gene therapy is to deliver therapeutic genes to treat disease. It is critical to identify safe and efficient gene therapy systems. Viruses are often used as efficient gene delivery vehicles, including adeno-associated virus (AAV). However, most viruses cannot be administered repeatedly as the body develops neutralizing antibodies that clear any subsequent treatments before they can deliver their genetic payload. This may be overcome by enveloping the AAV in an exosome coat. Exosomes are extracellular vesicles that carry signaling molecules between cells. Exosome:AAV (exo-AAV) formulations avoid the immune system and the development of antibodies, which increases our ability to target AAV to appropriate tissues and allow for repeated treatments. The student researcher will learn how to create and package these exo-AAVs, improve the purification systems, and test them in tissue culture systems. This research will have direct impact on developing a second generation of gene therapies based on enhancing the physical properties of the virus itself. 

Project outcome

The specific outcomes of this project will be identified by the faculty mentor at the beginning of your collaboration. 

Poster presentation, co-authorship on any publication generated.

Project details

Timing, eligibility and other details
Length of commitment Ideally the student will be able to commit at least two semesters
Start time Fall (August/September)
Summer (May/June) 
In-person, remote, or hybrid? Hybrid Project 
Level of collaboration Small group project (2-3 students) 
Benefits Academic credit
Other: Possible stipend support 
Who is eligible Sophomores
Juniors
Seniors 

Project mentor

Caroline Bass

Associate Professor

Pharmacology and Toxicology

Phone: (617) 304-8627

Email: cebass@buffalo.edu

Start the project

  1. Email the project mentor using the contact information above to express your interest and get approval to work on the project. (Here are helpful tips on how to contact a project mentor.)
  2. After you receive approval from the mentor to start this project, click the button to start the digital badge. (Learn more about ELN's digital badge options.) 

Preparation activities

Once you begin the digital badge series, you will have access to all the necessary activities and instructions. Your mentor has indicated they would like you to also complete the specific preparation activities below. Please reference this when you get to Step 2 of the Preparation Phase. 

learn how to write an annotated bibliography
attend all lab safety courses through EH&S
attend basic animal handling and orientation sessions through the Laboratory Animal Facility 

Keywords

pharmacology and toxicology