The Gene Therapy and Vaccines Program focuses on the use of gene transfer in animal models and humans, either for therapeutic purposes or for vaccination. The mission of the graduate program is to produce exceptional investigators who will become outstanding researchers in the field. Students in the program acquire a broad background in molecular biology, cell biology, physiology, immunology and virology. Research in the program includes basic cell biology, molecular biology, developmental biology, molecular physiology, virology and immunology. Although the goals of the research are disease-based with an ultimate objective directed to prophylactic and therapeutic applications, the research training focuses on basic investigations directed to understanding the pathobiology of relevant diseases and to achieving efficient and effective gene transfer in humans. Among the diseases of interest to program members are cystic fibrosis, hemophilia, lysosomal storage diseases, inherited blindness, cancer, cardiovascular diseases, and immunologic and infectious diseases. Research on vaccines for prophylactic as well as therapeutic indications are emphasized. Specific focus is provided through a survey course in gene therapy and vaccines and through advanced seminars in specific areas of gene therapy and/or vaccine research. In addition, the program runs a monthly seminar series featuring faculty members from Penn and other universities, as well as a bi-weekly trainee seminar where graduate students and post-doctoral fellows present their research.
See Academic of this site for more information on the CAMB graduate group's requirements and related topics.
(Click on links for course descriptions below.)
The following seminars are offered in Spring on a rotating basis:
Suggested Elective Courses:
CAMB 610: Molecular Basis of Gene Therapy
This is a team-taught, survey course that focuses on the basic science relevant to achieving efficient and effective gene transfer in animal models and humans for the treatment of disease. The course includes a unit devoted to a variety of vectors useful for gene transfer, with the remainder of the course devoted to the study of current gene therapy approaches using specific diseases as models. Prior background in biochemistry, cell biology, and molecular biology is essential. Aspects of organ system anatomy and physiology, virology and immunology that are relevant to the course material are included in the course. Because of the rapid movement in this field, specific topics vary somewhat from year to year. The course is designed for second year graduate students, however first year students may take the course with the course director's approval. Lecture format with discussion hours interspersed. There will be a take-home examination at the end of each of the three sections, each focusing on the material covered in that section. Offered fall semester. [up]
CAMB 609: Vaccines and Immune Therapeutics
Vaccination is the most successful medical technological intervention. The goal of this course is to expand on students' general understanding of the immune system and to focus this understanding towards the application of vaccination and immune therapies for the 21 century. Furthermore, the course will give the student a sense of how these principles are applied to vaccine and immune therapeutic development. The course covers basic science as well as the clinical, regulatory, ethical, and political issues and implications of modern vaccines and world health.
Initial lectures review immune mechanisms believed to be responsible for vaccine induced protection from disease. Subsequent lectures build on this background to explore the science of vaccines for diverse pathogens, including agents of bioterrorism as well as vaccines for cancer. An appreciation for the application of laboratory science to the clinical development and studies of vaccines is provided in the next section of the course along with lectures, which focus on the regulatory, safety, and ethical implications of vaccines in different world situations. The financial implications of specific vaccines on global health is one focus of the course.
The course is lecture style with many, many guest lecturers who are experts in their particular area of vaccine development. There are required readings to provide the student context and background for the diverse lectures topic. Students are graded on course participation, and a final project/exam. The project is to design in a powerpoint report a vaccine strategy for a current disease or pathogen of importance that does not as yet have an effective vaccine or immune therapy. Strategies used should build on the material presented in the class lectures. The course is intended for graduate students or medical students in various MS, Ph.D., or MD/Ph.D. programs on the campus, as well as local scientists and professionals in the community. As a prerequisite students should have taken biology, biochemistry, or immunology courses at the advanced college level.[up]
CAMB 510: Immunology for CAMB students
The purpose of this course is to provide a thorough grounding in immunology to Cell and Molecular Biology graduate students, with an emphasis on the role of the immune system in combating infectious and neoplastic disease, and its role in immunopathological states such as autoimmunity and allergy. This is a required course for CAMB students in the Microbiology, Virology and Parasitology program and the Vaccine and Gene Therapy program, replacing IMM 506 (Immune Mechanisms). It may also be used as an elective by CAMB students in other programs.
The course is divided into two parts. The first deals with basic innate and adaptive immune mechanisms, the structure, function, and molecular biology of antigen receptors and major histocompatibility complex molecules, and the development, activation, and differentiation of lymphocytes and other hematopoietic cells involved in immunity. The second part will cover the immune response to infection by bacteria, viruses, and parasites, and the clinical and applied aspects of immunopathological states such as cancer and autoimmunity. The course is comprised of two 1.5 hour lectures per week, and there will be two exams. The first will be taken after part I, and the second after part II of the course. Both will be open-book and in-class exams.[up]
The following seminars are offered on a rotating basis:
CAMB 633: Advanced Seminar in Gene Therapy
This year's Advanced Seminar in Gene Therapy will cover controversial topics in the field. It will meet on Wednesdays from 4:30 - 6:30pm. The goal of this seminar is to provide graduate students with an understanding of the challenges, both experimentally and practical, that face the gene delivery field. At least two sessions will deal with ethical issues. With the exception of the first class meeting, each of the weekly, two-hour sessions will be devoted to a discussion of two recent papers. All students are to have read the papers. Evaluation will be based on attendance (required), active participation, and preparation of reviews of papers. Students will be introduced to the process of manuscript review and will be asked to provide critical reviews for two manuscripts. Offered spring semester in odd years.
Students who are not in CAMB need to request permission from the course director, Dr. James Wilson, via email: firstname.lastname@example.org. [up]
CAMB 637. Gene Therapy: Vectors, Immunology and Disease
Prerequisite(s): Background in molecular biology, virology and immunology.
This seminar course is designed to provide students with a cohesive understanding of virology and immunology of gene therapy. Three major themes will be covered: vectors, vector immunology, and gene therapy of genetic and acquired diseases. The topics to be covered are viewed as an extension of topics covered in CAMB 610 (Molecular Basis of Gene Therapy), although CAMB 610 is not an absolute prerequisite for this seminar. Each class will consist of a brief introduction by an instructor, reviewing background information related to the theme discussion. The topics are explored through discussions, led by assigned students, of seminal research articles. Students are expected to have thoroughly reviewed the assigned articles and be able to present and discuss various aspects of the papers. Regular attendance and active participation in the discussions, which focus on critical evaluation of experimental design, data presentation and interpretation, is essential. Student evaluation will be based on attendance, in-class presentation (for 50% of the letter grade), and a take-home exam (for another 50% of the grade). Offered spring semester in even years. [up]
Suggested Elective Courses:
CAMB 548: Fundamental Virology
Prerequisite: Permission of instructor. A basic course in virology including molecular, cellular, immunological, and in vivo pathological aspects. Offered spring semester. [up]
CAMB 532: Human Physiology
This course aims to show how knowledge of cellular and molecular mechanisms can be integrated in an understanding of function at the tissue, organ and organism levels. It will begin with a description of the building blocks of a generic organ, a very brief survey of the major organ systems and their interrelationships, and an introduction to some of the specific techniques that will be discussed later. The bulk of the course, rather than attempting to be comprehensive, will explore selected topics in the areas of skeletal muscle, the heart and blood vessels, epithelial transport (kidney and GI tract) and endocrinology in moderate depth. It will use natural and experimental molecular defects and their phenotypes to illustrate integrated function. As well as forming the basis for the study of integrative physiology, it is intended for students of cellular and molecular biology and genetic engineering who will need to appreciate the roles of specific systems and molecules at higher levels of organization. Format: three 1.5 hour meetings per week, two for lecture/discussion, one for student presentations based on assigned key papers. Prerequisites: cell biology at the level of BIOM 600 (or alternate with the permission of the instructor), a general understanding of the chemistry and biochemistry of macromolecules, and of basic molecular biology. Offered fall semester. [up]