Gene therapy → → Gene therapy 1. Germline gene therapy 2. Somatic gene therapy Germline gene therapy – retrovirus vector Baby with genetic disease Parents with genetic disease Zygote with mutant gene Therapeutic gene Virus vector Treated culture Cell culture Embryo with genetic disease Enucleated egg cell Genetically modified Cell from the culture Genetically improved embryo Genetically improved zygote Healthy baby Somatic gene therapy in vivo gene therapy Virus vector Liposome Gene gun Somatic gene therapy ex vivo gene therapy virus vector liposome Somatic gene therapy in vivo gene delivery ex vivo gene delivery Genetically modified cells - Viral and other types of gene transfer Stem cell therapy: 1. Genetically non-modified stem cells 2. Genetically modified stem cells (gene therapy) Somatic gene therapy Direct (in vivo) gene delivery Cell-based (ex vivo) Gene delivery Therapeutic gene ES cells Therapeutic gene Retrovirus In vitro differentiated cells Adult stem cells Genetically modified cells Gene therapy of ADA David the bubble boy SCID (severe combined immunodeficiency disease): non-functioning B and T lymphocytes One of its type is caused by the mutation of ADA (adenosin deaminase) gene Earlier: bone marrow transplantation (bone marrow of relatives) Gene therapy: ADA gene delivery to lymphocyte by retrovirus vector Michael Kaplitt AAV-2 – GAD gene → AAV inoculation AAV vector for foreign gene foreign gene subthalamic nucleus receptor protein rekombinant AAV Secreted protein The first victim of gene therapy Jesse Gelsinger Ornithine transcarbamylase deficiency (X chr.-linked disease; playing a role in ammonia metabolism) Death was caused by the side effects of adenovirus vector The debate Do not interfere with the Creator’s business 9
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