introduction to gene therapy pdf

0000023970 00000 n The efficient transfer of the genetic material into a cell is necessary to achieve the desired therapeutic effect. 0000009693 00000 n 0 0000177415 00000 n (15 hr) § Introduction of fundamental immunology (3 hr) Two methods are available for inserting genetic material into human chromosomes. addition of -like globins or shRNAs 2. 0000012619 00000 n 0000062739 00000 n There is more than one way that gene therapy can be used. A virus is selected as a vector because of its ability to enter the body. 0000159866 00000 n 19 WHAT IS GENE THERAPY? 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�����. 0000040748 00000 n 227 73 0000051457 00000 n 0000010291 00000 n h�bbd```b``� 0000001756 00000 n 0000050869 00000 n 0000051757 00000 n 0000040016 00000 n 0000039147 00000 n 0000056301 00000 n 0000004902 00000 n 0000070984 00000 n 0000052400 00000 n 0000003686 00000 n 0000052031 00000 n A vector can be created by making changes to a naturally occurring virus. 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 0000055399 00000 n INTRODUCTION TO. Gene Addition –with integrating lentiviral vectors Universal for SCD and -thalassaemia E.g. 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. 0000070700 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) gene therapy clinical trials to da te ha ve involv ed ‘gene addition’. Gene therapy in the body often takes benefit of the natural inclination of viruses to infect certain organs. What are the different types of gene therapy? endstream endobj startxref Introduction of genetic material to heal or prevent disease ... Gene Therapy Approaches 1. 0000011484 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’. 0000003713 00000 n %PDF-1.7 %���� After … 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. 0000012202 00000 n mutation-specific precise repair 3. 0000009015 00000 n 0000051877 00000 n %%EOF ZoJn1�6�4�h`^�Q�U,D��u*���v�U���833p+p0�`�a���͠� |`�m���8�>��@�)iV��5 1��S�4#0 ʓ8 0000008423 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 0000005877 00000 n Gene therapy . A brief history of the development of gene therapies 3. Because blood-clotting agents can be attached to the blood in the liver, this virus is used in gene therapy to cure hemophilia. 0000006418 00000 n h�b``�g``�����lo� Ā B@16�S���$�t\ �y|!�!��@dX-0p\��:@��� Ȩ���������� ��7\F FM%��� 0000039969 00000 n 0000006165 00000 n 0000006800 00000 n Gene Correction –with designer nucleases or base editors Mutation-specific E.g. 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. 0000056568 00000 n The next article in the premium service will discuss the various delivery systems for gene therapy/editing. This gene then provides the instructions for your body to make the missing or insufficient protein. 0000000016 00000 n Outline Gene Therapy Principles Tools 0000050601 00000 n 0000019078 00000 n 0000007639 00000 n 0000002579 00000 n Genetic disease This type of disease is caused by a missing or nonworking gene or genes. 0000052231 00000 n 421 0 obj <>stream 0000125868 00000 n An introduction of what gene therapy is 2. 20 Gene therapy is the introduction of genetic material into a cell so that it produces a therapeutic 21 protein product. 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 0000024102 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 … 227 0 obj <> endobj xref INTRODUCTION 1. 1. Gene Therapy. 0000105919 00000 n 0000004541 00000 n 0000051227 00000 n 0000051603 00000 n Gene therapy Gene therapy is a methodology for correcting defective genes responsible for disease development. 0000011938 00000 n 305 0 obj <> endobj 0000013270 00000 n 0000009666 00000 n 0000041025 00000 n 0000003960 00000 n Over 60% of trials ha ve been for cancer, proba bly because of the large numbers of affe cted

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