GENE THERAPY

Within a Somatic Cell

• 3.2 billion base pairs

• 23 chromosomes pairs

• 30,000 genes

Gene Therapy

Products that harness the central dogma to modify genetic information and integrate into patients as viruses, genetically engineered microorganism, or nucleic acids

Monogenic Disease

Disease that results from changes/mutation to a single gene occurring in all cells of the body

• Sickle Cell Anemia

• Cystic Fibrosis

• Huntington Disease

• Hemophillia

Somatic vs Germ Gene Therapy

Somatic

• therapeutic genes transferred to somatic cells

• not inherited by later generations

• ex: Bone Marrow Cells, Blood Cells, Skin Cell

Germline

• therapeutic genes transferred to germ cells

• inherited by later generations

• ex: egg and sperm

Crietias of Gene Theapry

• Must be effective therapeutic genes that can be expressed at speflic target sites ( advangtes to disease state of patient )

• Must be efficient and safe delivery system ( not effecting other cells/processes )

Adenovirus Vector

Non-pathogenetic form of an virusis that is packaged with modified DNA in which vector binds onto membrane of cell for entry

• Vector once in cell is packaged with vesicle that break down once reaches nucleus to inject new gene within nucleus to allow for the creation of proteins using new gene

Retrovirus Vector

A modifided retroviruis RNA that is recombinated with human RNA and reverse transcriptase

• Once entry into cell reverse transicrption can occur between DNA complemnarty strand and recombinant RNA to create DNA ( which is randomly inserted into genome

In Vivo vs Ex Vivo

In Vivo

• theapeaturic gene is cloned into recombinant virus genome that is infused into patient cells which are transduced to produce therapeutic proteins

  • less invasie, safety check not possible, decreased control, technically simple, vector introduced directly

Ex Vivo

• cells from patient are extracted through biopsy and expanded in cell culture to be transduced by recombinant virus with therapeutic gene to create tranduced cells that can create therapeutic protein to be infused back to patient

  • more invasie, technically complex, safety check allowed, more control, no vector direct contact

Immunotherapy

Treatment of harnessing a person immune system to fighting disease

• Can be done by either stimulating own immune system to work harder/attacker smarter or provide immune system with extra components

Cytotoxic T Cell

Contains perform and granzymes which form pores on target cell when binded to release materials within target cell to kill it

CAR-T Cell Therapy

Patient T-Cells are removed and genetically modified ex-vivo to attack cancer cells

• CD-19 is a marker outside B-Cells, especially within cancerous B-Cells such as leukemia and lymphoma which CAR-T can be used for to idefinty B-Cells

Retina

Layer in the back of the eye that contains light sensitive cell that trigger nerve impulse from optic nerve to brain to create image

• Retinal Pigment Epithelium behaves as barrier to retina providing protection/stability

Epidermolysis Bullosa

A condition an individual skin is very delicate and break off very easily

• Serval forms of this disease which can result from serval genes