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Advances in Gene Therapy

(2 hours of credit)
 Ronald Klein, Ph.D., Course Director

Text: Understanding Gene Therapy
Nicholas R. Lemoine (Editor)

Schedule – TBA
Location – TBA
1hr/lecture

Lecture Number

Lecturer

Topic

1, 2

Klein

History of gene therapy and related clinical trials
Book Chapters 1, 2, 9

3, 4

Klein

Nonviral and viral vectors
Book Chapters 3, 4

5, 6

Ryman

RNA viruses

7, 8

Mathis

Adenovirus vectors

9, 10

Klein

Adeno-associated virus vectors

11, 12

Turturro

Vector targeting
Book Chapter 8

3 hr

Klein

Student oral presentations

2 hr

 

Mid-term Exam

13, 14

Williams

Blocking gene expression

15, 16

Zhang

Immune responses to vectors and transgene products

17, 18

Duan

Cell-based therapies

19, 20

Turturro

Single gene and multifactorial disorders
Book Chapters 5, 6, 7

20, 21

Williams

Cancer gene therapy

21, 22

Klein

CNS gene therapy

23, 24

Mathis

Gene therapy bioethics
Book Chapters 10, 11

3 hr

Klein

Student oral presentations

2 hr

 

Final Exam

Catalog description: An overview of gene therapy emphasizing the clinical history, the types of diseases that could benefit the most, and ethical issues.  Some of the major gene transfer vector systems will be covered in detail, highlighting advances in this rapidly developing field.  Students’ grades will be based on participation in discussion of current research and review articles, as well as exams.

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