Gene therapy training: CF and Genetic Diseases

描述（由申请人提供）：这是一份申请，申请继续资助与囊性纤维化和其他遗传疾病相关的基因治疗基础科学培训。该培训项目设在宾夕法尼亚大学人类基因治疗研究所（IHGT），该研究所的教育使命是培养将成为该研究领域领导者的杰出研究人员。该项目前5年共培养1名博士生和3名博士后，培养了2名博士生和7名博士后。在此期间，新建立的基因治疗研究生培训项目蓬勃发展；因此，现申请支持3个博士预科和3个博士后职位。博士预科学员将是生物医学研究生（BGS）项目的学位论文水平的学生，该项目是宾夕法尼亚大学生物医学科学教育的管理机构，可以是博士学位学生，也可以是综合学位学生。大多数获得培训资助的学生将被BGS的细胞和分子生物学（CAMB）研究生组录取，基因治疗是其六个项目之一。在其他研究生组从事与遗传疾病基因治疗相关的论文项目的学生也将符合条件。博士后培训生将具有博士学位或医学博士学位。，或同等学位，并将在项目教师的实验室进行基础研究培训。所有职位的候选人将由个别项目教员提名。被提名者将由培训资助教师委员会进行评估和排名，最合格的申请人将获得支持。在整个培训过程中，所有学员都将参加IHGT系列研讨会、IHGT年度退修会和基因治疗培训生系列研讨会。他们还将参加与其实验室主要研究兴趣相关的研究研讨会和期刊俱乐部。所有受训者都将被要求学习由该培训项目开发的“基因治疗分子基础”课程，以及有关研究伦理行为、实验动物的护理和使用以及化学、生物和辐射安全的课程。将支持受训者出席国家会议，介绍其工作成果。培训教员是拥有良好支持的基础科学实验室的知名研究人员，他们正在开发针对遗传病的基因疗法。

DESCRIPTION (provided by applicant):This is an application for continued funding for training in the basic sciences of gene therapy related to cystic fibrosis and other genetic diseases. The training program is based in Penn's Institute for Human Gene Therapy (IHGT) whose educational mission is to produce exceptional investigators who will become leaders in this field of research. In its first five years, this NIDDK-funded training program had positions for one predoctoral and three postdoctoral fellows, which supported two predoctoral and seven postdoctoral trainees. During this period, a newly established graduate training program in gene therapy has flourished; thus support is now requested for three predoctoral and three postdoctoral positions. Predoctoral trainees will be dissertation-level students in the Biomedical Graduate Studies (BGS) program, which is the governing body for biomedical science education at Penn, either as Ph.D. or combined degree students. Most students supported by this training grant will be enrolled in the Cell and Molecular Biology (CAMB) graduate group of BGS, in which Gene Therapy is one of its six programs. Students working on dissertation projects related to gene therapy of genetic diseases in other graduate groups will also be eligible. Postdoctoral trainees will hold either a Ph.D. or M.D./Ph.D., or equivalent degrees and will be trained in basic research in the laboratories of the program faculty. Candidates for all positions will be nominated by individual program faculty members. Nominees will be evaluated and ranked by a committee of the training grant faculty, and the most qualified applicants will be offered support. Throughout the training program, all trainees will attend the IHGT seminar series, the IHGT annual retreat, and the trainee seminar series in gene therapy. They will also participate in research seminars and journal clubs relevant to their laboratory's primary research interest. All trainees will be required to take "Molecular Basis of Gene Therapy," a course that was developed by this training program, as well as courses in the ethical conduct of research, care and use of experimental animals, and chemical, biological and radiation safety. Trainees will be supported to attend national meetings to present the results of their work. The training faculty are established researchers with well supported basic science laboratories developing gene therapy for genetic diseases.