Germline gene therapy

Gene therapy

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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

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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

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