0000010872 00000 n 0000052612 00000 n 0000005877 00000 n 0000005914 00000 n 0 0000018992 00000 n ZoJn1�6�4�h`^�Q�U,D��u*���v�U���833p+p0�`�a���͠� |`�m���8�>��@�)iV��5 1��S�4#0 ʓ8 trailer <]/Prev 507543>> startxref 0 %%EOF 299 0 obj <>stream 0000011938 00000 n 1. There are two types of gene therapy, which differ in various ways, as shown in the table below. 0000052231 00000 n INTRODUCTION 1. mutation-specific precise repair 3. How are the genes changed with gene therapy? 0000050869 00000 n INTRODUCTION Currently, gene therapy refers to the transfer of a gene that encodes a functional protein into a cell or the transfer of an entity that will alter the expression of an endogenous gene in a cell. 0000125868 00000 n 0000012619 00000 n 227 73 0000000016 00000 n Brief Introduction To Gene Therapy Oct. 28, 2019 12:53 PM ET Bhavneesh Sharma Registered investment advisor, biotech, contrarian Summary This is the first article in a series on gene therapy/editing which was first published on the Marketplace premium service last week. Gene Correction –with designer nucleases or base editors Mutation-specific E.g. endstream endobj startxref 0000056301 00000 n 0000159866 00000 n 0000051757 00000 n Gene therapy is a technique that uses genetic material (a piece of DNA) for the long-term treatment of genetic disorders.1,2 This may involve delivering a copy of a healthy or therapeutic gene, repairing a faulty gene, and/or altering the degree to from the body) which a gene is turned ‘on’ and ‘off’. Adeno-associated virus, for instance, runs directly to the liver when it is introduced into the bloodstream. What are the different types of gene therapy? 0000039969 00000 n 0000052031 00000 n 0000051457 00000 n Gene therapy Gene therapy is a methodology for correcting defective genes responsible for disease development. • Gene therapy for genetic diseases • Gene therapy for infectious diseases • Gene therapy for cancer (2 hr) • Gene therapy clinical trials • Part 1 literature presentation and discussion • Examination 1 Part 2: Introduction of immunology and immunotherapy and applications. Two methods are available for inserting genetic material into human chromosomes. (15 hr) § Introduction of fundamental immunology (3 hr) Over 60% of trials ha ve been for cancer, proba bly because of the large numbers of affe cted 0000024172 00000 n Gene replacement therapy starts with scientists creating a new, working copy of a missing or nonworking gene. 0000039495 00000 n 0000009152 00000 n 0000041025 00000 n 0000008423 00000 n Introduction to Gene and Cell Therapy David Barrett, JD Executive Director, ASGCT. 0000005355 00000 n endstream endobj 306 0 obj <>/Metadata 20 0 R/OpenAction 307 0 R/PageLayout/SinglePage/Pages 303 0 R/StructTreeRoot 49 0 R/Type/Catalog/ViewerPreferences<>>> endobj 307 0 obj <> endobj 308 0 obj <>/Font<>/ProcSet[/PDF/Text/ImageC]/Properties<>/Shading<>/XObject<>>>/Rotate 0/StructParents 0/TrimBox[0.0 0.0 595.276 595.276]/Type/Page>> endobj 309 0 obj <>stream A vector can be created by making changes to a naturally occurring virus. A gene therapy must therefore consist of genetic information, the transgene, and a 22 way of introducing it into the cell, the vector. 0000040016 00000 n However, since hetero- zygotes with 50% of normal gene product can be severely affected, successful gene therapy for heterozygotes requires efficient expression of the introduced genes. h�bbd```b``� 0000055399 00000 n 421 0 obj <>stream 0000003686 00000 n
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