Department of Pediatrics

College of Medicine

University of Florida

 

GMS 6251: Molecular Therapy I – Vectors and Molecular Mechanisms

 

Syllabus

Fall 2016 (even years)

                                                                                                                                                                         

Class Meetings:          TBD (Fall B)

Class Location:          Cancer and Genetics Research Complex, Room 436                                    

Credit Hours:            1 credit (3, 1-hour sessions per week for 5 weeks)

 

Course Director:       Sergei Zolotukhin, PhD

Office Hours:                         TBD

Office:                                    CGRC, room 237

 

Phone:                        352-273-8150

Website:                     TBD

                                                                                                                                                                                                          

           

COURSE Description

Gene therapy has become an established molecular medicine for treatment of multiple genetic diseases (such as primary immune deficiencies, inherited forms of blindness, and others) and certain types of cancer. The University of Florida has one of the most renowned gene therapy centers. This course covers the most important/widely studied diseases and applications of gene therapy. Particular importance is given to those diseases successfully treated by gene therapy in clinical trials.

 

Course Objectives
Students who successfully complete the course will be able to:

• demonstrate a basic understanding of which diseases are amendable to treatment by gene therapy and why;
• understand the advantages gene therapy may offer over other treatment modalities for a specific disease;
• distinguish between in vivo and ex vivo gene therapy;
• understand the principles and mechanism of disease correction by gene transfer;
• understand the basis of several clinical gene therapy protocols;
• appreciate the varying challenges of gene therapy for different diseases and target organs
• relate development of gene transfer technologies to treatment of disease
• gain a basic understanding of how clinical trials in gene therapy are regulated

 

Course Procedure

This graduate-level course is intended to provide students with an up-to-date knowledge of gene therapy for human disease; to introduce the students to cutting edge approaches to clinical gene therapy; and to understand the basic science that underlying development of gene therapies for different diseases and target cells/organs. The teaching format is a mixture of formal lectures and student presentation. Each topic is taught by a different instructor, who is an expert on the specific disease or approach. During those sessions dedicated for presentations, two students will give a power point presentation of 20 min, discussing an assigned research paper on the topic, followed by 10 min group discussion. Each student will have to give at least one such presentation. The proposed schedule is designed for 8 presentations. If more students enroll in the class, the schedule will be adjusted accordingly. A summary discussion and a final exam will conclude the class. Finally, this class will prepare students for the courses Molecular Therapy II and III.

 

Student Course Requirements

The following will be used to assess students’ progress in achieving the course objectives:

 

1. Reading assignments and Oral Presentation. At the beginning of the course, review articles will be provided to the students as background information, and the students will be alerted to the relevant chapters in the text book that accompany each topic. In addition, the students will be given pdf files of all original research and a schedule of when each paper will be discussed. During those sessions dedicated for presentations, two students will give a power point presentation of 20 min, each discussing an assigned research paper on the topic, followed by 10 min group discussion. Each student will have to give at least one such presentation. However, all students are required to have thoroughly read and analyzed both papers prior to each session and to participate in discussion.

 

2. Attendance and Participation. Each student is expected to be an active and regular participant in all class discussions.

  

3. Final exam. The final exam will be a mixture of multiple choice and short answer questions and cover all topics discussed in lecture format during the course.

 

EVALUATION AND GRADING

Grades will be based on attendance (20%); quality and degree of participation in discussions (10%); quality of the oral presentation (30%) and the grade resulting from the final exam (40%).  The grading scheme will be as follows:

 

Grading scheme

 

A      (outstanding)                  93-100

A-     (superior)                        90-92

B+    (excellent)                      87-89

B      (very good)                    83-86

B-     (above average)              80-82

C+    (average)                        77-79

C      (slightly below average) 73-76

C-     (below average)              70-72

D+    (well below average)      67-69

D      (poor)                             63-66

D-     (very poor)                     60-62

E       (failure)                          <60

 

 

Textbook and Readings:

The following textbook is required for this course:

 

Herzog RW and Zolotukhin S (2010) A guide to human gene therapy. World Scientific, 377 pp.

 

In addition, articles from biomedical journals will be assigned at the beginning of the class (2 paper per oral presentation session). Finally, review articles on gene therapy will be provided to the students.

 

 

Topical Overview

 

Week	Major Topic	Type of class	Date	Instructor
1	Gene transfer – introduction and overview	Lecture	August, 22	Sergei Zolotukhin
2	Plasmid-based vectors and nanoparticles	Lecture	August, 24	Bradley Fletcher
3	Transposons and integration vectors	Lecture	August, 26	Bradley Fletcher
4	RNA technologies I	Lecture	August, 29	Alfred Lewin
5	RNA technologies II	Student presentations	August, 31	Alfred Lewin
6	Gene-modified stem cells I	Lecture	September, 2	Sihong Song
7	Gene-modified stem cells II	Student presentations	September, 7	Sihong Song
8	Retroviral and Lentiviral vectors	Lecture	September, 9	David Marcusic
9	Adenoviral vectors	Lecture	September, 12	Nicholas Muzyczka
10	CRISPER Cas9	Lecture	September, 14	Edgardo Rodriguez
11	Structure-based vector engineering	Lecture	September, 16	Mavis Agbandje-McKenna
12	Adeno-associated viral vectors I	Lecture	September, 19	Sergei Zolotukhin
13	Combinatorial/rational approaches in vector design	Lecture	September, 21	Sergei Zolotukhin
14	Adeno-associated viral vectors II	Student presentations	September, 23	Sergei Zolotukhin
15	Exam	Exam	September, 26	Sergei Zolotukhin

 

 

Course Policies

 

Students are expected to adhere to the following course policies.

 

Course Website

Additional detail, regular updates to the course schedule and assignments, and announcements will be posted on the course web site.  You are expected to check the web site on a regular basis (i.e., at least one day prior to each class meeting).

 

Class Decorum

Please: (1) be on time, (2) respect others’ points of view, (3) listen quietly when others are speaking, and (4) turn off cell phones, alarms, and other such distractions.

 

Returned Assignments

Keep copies of all assignments that you submit and of all grades until you receive official notification of your final course grade.

 

Attendance Policy 

Class attendance is mandatory. Excused absences follow the criteria of the UF Graduate Catalog (e.g., illness, serious family emergency, military obligations, religious holidays), and should be communicated to the instructor prior to the missed class day when possible. The UF Graduate Catalog is available at http://gradcatalog.ufl.edu/.  Students should read the assigned readings prior to the class meetings, and be prepared to discuss the material.  Regardless of attendance, students are responsible for all material presented in class and meeting the scheduled due dates for class assignments.

 

Policy on Make-Up Work 

Students are allowed to make up work only as the result of illness or other unanticipated circumstances or for religious exemptions.  In the event of such emergency, documentation will be required in conformance with university policy.  Work missed for any other reason will earn a grade of zero.

 

Special Needs

Students requiring accommodations must first register with the Dean of Students' Office.  The Dean of Students' Office will provide documentation to the student who must then provide this documentation to the faculty member when requesting accommodation. The College is committed to providing reasonable accommodations to assist students in their coursework.

 

Academic Honesty

Each student is bound by the academic honesty guidelines of the University and the student conduct code printed in the Student Guide and on the University website, available at http://regulations.ufl.edu/chapter4/4017.pdf).  The Honor Code states: “We, the members of the University of Florida community, pledge to hold ourselves and our peers to the highest standards of honesty and integrity."  Cheating or plagiarism in any form is unacceptable and inexcusable behavior.

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Policy on Style for Citation and Plagiarism:

The two key purposes of citation are to (1) give appropriate credit to the authors of information, research findings, and/or ideas (and avoid plagiarism) and (2) facilitate access by your readers to the sources you use in your research.

 

Quotations: When directly quoting an outside source, the borrowed text, regardless of the amount, must be surrounded by quotation marks or block quoted. Quoted text over two lines in length should be single-spaced and indented beyond the normal margins. Every quote must include a source—the author, title, volume, page numbers, etc.—whether an internal reference, footnote, or endnote is used in conjunction with a bibliography page.   

 

Paraphrasing or Citing an Idea: When summarizing an outside source in your own words or citing another person’s ideas, quotation marks are not necessary, but the source must be included.  This includes, but is not confined to, personal communications from other students, faculty members, experts in the field, summarized ideas from published or unpublished resource, and primary methods derived from published or unpublished sources.  Use the general concept of “when in doubt – cite.”

 

Plagiarism is a serious violation of the academic honesty policy of the College. If a student plagiarizes others’ material or ideas, he or she may receive an “E” in the course. The faculty member may also recommend further sanctions to the Dean, per College disciplinary action policy. Generally speaking, the three keys of acceptable citation practice are:  1) thoroughness, 2) accuracy, and 3) consistency.  In other words, be sure to fully cite all sources used (thoroughness), be accurate in the citation information provided, and be consistent in the citation style you adopt.  All references should include the following elements: 1) last names along with first and middle initials; 2) full title of reference; 3) name of journal or book; 4) publication city, publisher, volume, and date; and 5) page numbers referenced.  When citing information from the Internet, include the WWW address at the end, with the “access date” (i.e., when you obtained the information), just as you would list the document number and date for all public documents. When citing ideas or words from an individual that are not published, you can write “personal communication” along with the person’s name and date of communication.