유전자치료공학

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유전자치료공학

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페이스북 트위터 카카오톡 네이버밴드 링크복사

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자연과학 >생물ㆍ화학ㆍ환경 >생명공학
강의학기

2017년 1학기

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강의계획서: 강의계획서

유전자 이상으로 생기는 질병을 DNA 또는 RNA를 사용하여 치료하려는 응용학문이다.

DNA 또는 RNA를 이용하여 질병을 일으키는 단백질의 과발현을 억제하거나, DNA 에디팅을 통하여 유전자이상을 고치는 방법 등을 학습한다.

수강안내 및 수강신청
※ 수강확인증 발급을 위해서는 수강신청이 필요합니다

Genetic Pharmacology I

차시별 강의

1.	Genetic Pharmacology I	1. Antigene Strategy 2. Antisense Strategy
	Genetic Pharmacology II	2. Antigene Strategy 3. Delivery Issues
2.	Principles of RNAi	1. Principles of RNAi Trigger Expression for Gene Therapy
	Principles of RNAi	2. On Demand Alternative Splicing for Gene Rescue 3. Nuclease-Mediated Targeted Genetic Correction
3.	Small regulatory RNAs	1. Therapeutic agents based on ribonucleic acid 2. small interfering RNAs (siRNAs)
	Small regulatory RNAs	3. microRNAs mimics (miRNA mimics)
4.	Gene Delivery	1. Cellular varriers to gene delivery 2. Endocytosis
	Gene Delivery	3. Escaped from the intracellular vesicle compartment 4. Direct inoculation of DNA and RNA 5. Sonoporation, microparticles, Liposomes, cationic polymers
5.	CRISPR/Cas9 Genome Engineering	1. Origin of CRISPR/Cas system 2. Application of CRISPR/Cas system 3. Major components of CRISPR/Cas9 genome editing system 4. Mechanism of CRISPR/Cas9 genome editing 5. Delivery of CRISPR/Cas9 6. Advantages of CRISPR/Cas9 genome editing
	Vectors and Gene Delivery Techniques 1	1. Vectors (Viral vector) 2. Retrovirus- and Lentivirus-Derived Vectors for Gene Transfer and Therapy
6.	Vectors and Gene Delivery Techniques 1	2. γ-Retrovirus- and Lentivirus-Derived Vectors for Gene Transfer and Therapy 3. Adenovirus vectors
	Vectors and Gene Delivery Techniques 1	3. Adenovirus, Adeno-Associated Virus (AAV) Vectors 4. Herpes Simplex Virus (HSV-1)-Based Vectors: Applications for Gene Transfer, Gene Therapy, Cancer Virotherapy and Vaccination
7.	Vectors and Gene Delivery Techniques 2	1. Vectors (Non-viral vector) 2. Macromolecular Conjugates for Non-Viral Nucleic Acid Delivery 3. Auto-Associative Lipid-Based Systems for Non-Viral Nucleic Acid Delivery 4. Electrotransfer/Electroporation for Non-Viral Nucleic Acid Delivery
8.	Therapeutic Applications 1	1. Oncolytic Adenoviruses for Cancer Gene Therapy
9.	Therapeutic Applications 1	2. Progress in DNA Vaccine Approaches for Cancer Immunotherapy
10.	Therapeutic Applications 2	1. Gene Therapy of the Beta-Hemoglobinopathies 2. Gene Therapy for Hemophilia A and B
	Therapeutic Applications 2	3. Experimental and Clinical Ocular Gene Therapy 4. Gene Therapy of Neurological Diseases
11.	Gene Vector Production	1. Production and Purification of Viral Vectors
	Gene Vector Production	2. Safety Considerations Related to Gene Vector Production
12.	Clinical application of gene therapy: General considerations	1. Number/ phases of clinical trials 2. Disease addressed by gene therapy clinical trials 3. Therapeutic genes used in the clinical trials 4. Modality of therapeutic gene delivery in gene therapy clinical trials
13.	Ethical and social problems of gene therapy	1. Safety of clinical experimentation 2. Ethical and social problems of gene therapy 3. Regulation and guidelines of gene therapy