遗传病的治疗课件.ppt

* * 加标题 * * DNA transfer into cells DNA转移到细胞 Ideal vector: safe, easily introduce, life-long expression 安全，容易引入，终身表达 Types: Viral vector nonviral vectors 病毒载体或非病毒载体 Viral Vectors 病毒载体 Types: Retroviruses 逆转录病毒 Adenoviruses AV腺病毒 Adeno-associated viruses AAV腺相关病毒 Nonviral Vectors 非病毒载体 Four general types: Naked DNA 裸露DNA DNA packed in Liposomes 脂质体包裹的DNA Protein-DNA conjugates 蛋白质-DNA融合体 Artificial chromosome 人工染色体 Risk of Gene Therapy 风险性 Adverse response to the vector or vector-disease combination 对带菌疾病的不良反应 Insertional mutagenesis causing malignant neoplasia 插入突变导致恶性肿瘤 Insertional inactivation of an essential gene 插入致重要基因失活 Some Candidate Diseases for Gene Therapy 一些基因治疗的候补 Clinical cases of gene therapy Adenosine Deaminase deficiency ADA deficiency 腺苷酸脱氢酶缺陷 French Anderson (NIH) LTR ADA SV40 neo LTR September 14, 1990 Retrovirus (Mo-MLV) T cell Gene Therapy Trials Ashanti de Silva Ashanti was the first patient to be treated with gene therapy. （她是第一个基因疗法成功的病例） Injections repeated 7 times in first 10.5 months ADA: 1% 25% Jesse Gelsinger: 18 yr-old from Arizona, died on 9/17/1999, 4 day after gene transfer in Penn OTCD trial. 这家伙就惨了，这位治疗失败 Gelsinger Case Tools of Human Molecular Genetics人类分子遗传学工具 Shujuan Song Department of Medical Genetics TO MASTER: Methods to isolate large quantities of a particular desired DNA sequence of interest分离出大量的特定DNA序列的方法 Methods of nucleic acid analysis 核酸分析的方法 The Ploymerase Chain Reaction PCR技术 The use of molecular cloning and the PCR to isolate large quantities of a particular desired DNA sequence of interest in pure form METHODS OF NUCLEIC ACID ANALYSIS Southern Blotting S-BLOT Analysis with Allele-Specific Oligonucleotide (ASO) Probes分析与等位基因特定寡核苷酸探针 Northern Blotting N-BLOT The southern blotting procedure Detection of single base-pair mutations using ASO probes The Ploymerase Chain Reaction PCR: enzymatic amplification of a fragment of DNA located between a pair of pr