0000054046 00000 n 0000004541 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’. 0000003686 00000 n %PDF-1.7 %���� 0000012202 00000 n 0000002448 00000 n Gene replacement therapy This type of gene therapy uses a new, working gene to replace the function of a nonworking or missing gene. 0000177415 00000 n Then the new gene is placed inside a vector, which acts like an envelope and carries the gene to the right places throughout the body. Introduction to Gene and Cell Therapy David Barrett, JD Executive Director, ASGCT. • 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. After … 0000051757 00000 n Gene Correction –with designer nucleases or base editors Mutation-specific E.g. 0000024172 00000 n An explanation of how gene therapy works 4. 20 Gene therapy is the introduction of genetic material into a cell so that it produces a therapeutic 21 protein product. INTRODUCTION TO. 0000052231 00000 n 0000052400 00000 n 305 0 obj <> endobj 0000005355 00000 n addition of -like globins or shRNAs 2. 0000011484 00000 n A virus is selected as a vector because of its ability to enter the body. 0000051457 00000 n 227 0 obj <> endobj xref 0000159866 00000 n 0000012619 00000 n Introduction to Gene & Cell Therapy for Haemoglobinopathies The Cyprus Institute of Neurology and Genetics 3 August 2020 Virtual Teaching Programme Lecture ARISE African Research and Innovative Initiative for Sickle Cell Education. A brief history of the development of gene therapies 3. 0000024102 00000 n Introduction of genetic material to heal or prevent disease ... Gene Therapy Approaches 1. 0000033942 00000 n 0000050869 00000 n 0000039147 00000 n 0000041324 00000 n 0000003846 00000 n 19 WHAT IS GENE THERAPY? Over 60% of trials ha ve been for cancer, proba bly because of the large numbers of affe cted 0000055399 00000 n This gene then provides the instructions for your body to make the missing or insufficient protein. Gene therapy in the body often takes benefit of the natural inclination of viruses to infect certain organs. 1. Two methods are available for inserting genetic material into human chromosomes. Genetic disease This type of disease is caused by a missing or nonworking gene or genes. 0000051603 00000 n gene therapy clinical trials to da te ha ve involv ed ‘gene addition’. 0000018992 00000 n 0000139917 00000 n 0000090933 00000 n Gene therapy . 0000006165 00000 n %%EOF A vector can be created by making changes to a naturally occurring virus. 0000052612 00000 n 0000003713 00000 n 0000040748 00000 n 0000052031 00000 n (15 hr) § Introduction of fundamental immunology (3 hr) gene therapy clinical trials to da te ha ve involv ed ‘gene addition’. A gene therapy must therefore consist of genetic information, the transgene, and a 22 way of introducing it into the cell, the vector. 0000013873 00000 n 421 0 obj <>stream INTRODUCTION 1. h�b```b``�g�``0=� Ā Bl�,.��\S���]+�¶����}��ֆ�2[��v` p21���x6�r+�Lܼ>hAY�'���K��v��'�l�m^���Ff�BeV��)3����j��|��>��NS�����m�e�s��t�K��MRr�(��-n�����. Adeno-associated virus, for instance, runs directly to the liver when it is introduced into the bloodstream. 0000070984 00000 n Starting with the basics • Genes are made up of DNA, which are the blueprints to build the enzymes or proteins that perform various crucial bodily functions. %PDF-1.5 %���� 0000054501 00000 n 0000006418 00000 n 0000105919 00000 n 0000013270 00000 n 0000005877 00000 n 0000002579 00000 n The efficient transfer of the genetic material into a cell is necessary to achieve the desired therapeutic effect. 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. Outline Gene Therapy Principles Tools An introduction of what gene therapy is 2. 0000011938 00000 n 0000009693 00000 n 0000050640 00000 n 0000051877 00000 n 0000010291 00000 n 0000009015 00000 n Introduction to Gene and Cell Therapy ... development time for gene therapy **Sometimes the three phases of a clinical trial are combined \⠀愀渀搀 昀漀爀 攀砀瀀攀搀椀琀攀搀 瀀愀琀栀眀愀礀猀尩 to make the evaluation process mor\൥ efficient, especially for serious and … 0000008423 00000 n 227 73 0000039448 00000 n 0000003960 00000 n 0000040016 00000 n ZoJn1�6�4�h`^�Q�U,D��u*���v�U���833p+p0�`�a���͠� |`�m���8�>��@�)iV��5 1��S�4#0 ʓ8 mutation-specific precise repair 3. What are the different types of gene therapy? If a dominantly inherited disorder is the result of a loss-of function mutation, treatment may be possible using conventional gene augmentation therapy. 0000053554 00000 n Gene Therapy. 0000051227 00000 n Gene Addition –with integrating lentiviral vectors Universal for SCD and -thalassaemia E.g. 0000050601 00000 n 0000023970 00000 n Over 60% of trials ha ve been for cancer, proba bly because of the large numbers of affe cted 0000041025 00000 n 0000007060 00000 n There are two types of gene therapy, which differ in various ways, as shown in the table below. endstream endobj startxref 0000009666 00000 n 0000125868 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��Z�r�8~�}�ܩ����`�T�l��lb'ce'���#�7�!�Ğ��n��e]�O�h4A����� i�%��Ԋ��`����HG�3�P�e*�����LĴ��3�5���\b+Y'�"?�i�fB#k�&Tl��LXI=8�ǜ��K���MB�b��H��L��(�D��� �Z�Zq����qEQ��E-�K�gڒ. 0000056568 00000 n 352 0 obj <>/Filter/FlateDecode/ID[]/Index[305 117]/Info 304 0 R/Length 186/Prev 588216/Root 306 0 R/Size 422/Type/XRef/W[1 3 1]>>stream 0000010872 00000 n 0000004902 00000 n There is more than one way that gene therapy can be used. 0000039495 00000 n 0000005914 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. 0000007639 00000 n Gene replacement therapy starts with scientists creating a new, working copy of a missing or nonworking gene. A summary of where gene therapy research is today which includes: current challenges, examples of advances with gene therapy treatments, and what the future might hold Gene Therapy Interactive 0000062739 00000 n Introduction to Gene Therapy: A Clinical Aftermath In both studies, viral shedding and increases in neutralizing antibodies were obser ved, but no serious adverse event ( 8 ) was h�bbd```b``� 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) trailer <]/Prev 507543>> startxref 0 %%EOF 299 0 obj <>stream 0000019078 00000 n Because blood-clotting agents can be attached to the blood in the liver, this virus is used in gene therapy to cure hemophilia. 0000001756 00000 n 0000000016 00000 n 0000006800 00000 n 0000031293 00000 n h�b``�g``�����lo� Ā B@16�S���$�t\ �y|!�!��@dX-0p\��:@��� Ȩ���������� ��7\F FM%��� 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

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