2.2 MedInt PLTW

Unit 2.2 discussing gene therapy & ethics

Gene Therapy

a type of disease treatment in which faulty genes are replaced by functional copies.

Vectors

DNA delivery systems. Typically a virus or plasmid

Cloning

Using a somatic or body cell from a multicellular organism to make one or more genetically identical individuals

In Vitro Fertilization

A procedure in which gametes are fertilized i a dish in the labratory, and the resulting zygote is implanted in the uterus for development

Preimplantation Genetic Diagnosis (PGD)

In assistede reproductive technology, the determination of genetic abnormalities in the embryo before it is transferred to the uterus

Sex Selection

Methods used to predetermine or diagnose the sex of an embryo for the purpose of seecting only those embryos of a particular sex for transplanting to the uterus of a woman

Faulty genes

The cause of many devistating illnesses & disorders

Genome editing

A technique that uses tools in the cell to cut DNA & replace mutated genes. The process can remove viral genes that have been inserted in the human genome & cause infectious diseases. Can be used to alter the DNA in stem cells.

Stem cells

Specialized cells that have the ability to develop into many different types of cells.

In Vivo therapy

The introduction of a vector directly into a patient

In Vitro therapy

The extraction of patient cells, then introduction of a vector into those cells, and then the reintroduction of those cells into the patient.

Hemophilia

A type of genetic disorder resulting in faulty clotting agents.

F8 Gene

Located on the X-chromosome. Codes for the liver cells that make clotting agents.

How many base pairs does gene F8 contain?

2,600 bp

CRISPR

(Clustered Regularly Interspaced Short Palindromic Repeats) A form of gene therapy

Cas-9

A CRISPR-associated protein and enzyme that acts as molecular scissors to cut DNA at specific locations specified by guide RNA.

Plasmid (Vector type)

• Carries any size gene

• Carries DNA

• No cell specificity

• Does NOT trigger immune response

• Is NOT integrated into the host cell genome

• Less efficient

Liposome (Vector type)

• Carries any size gene

• Carries DNA

• No cell specificity

• Does NOT trigger immune response

• Is NOT integrated into the host cell genome

• Less efficient

Herpes Virus (Vector type)

• Carries up to 20,000 base pairs

• Carries DNA

• Specific to nervous system

• triggers immune response

• Is NOT integrated into the host cell genome

• Less efficient

Adeno-associated Virus (AAV)

• Carries up to 5,000 base pairs

• Carries DNA

• No cell specificity

• Does NOT trigger immune response

• Is integrated into the host cell genome

• efficient

Adenovirus

• Carries up to 7,500 base pairs

• Carries DNA

• No cell specificity

• triggers immune response

• Is NOT integrated into the host cell genome

• efficient

Retrovirus

• Carries up to 8,000 base pairs

• Carries DNA

• Specific to dividing cells

• triggesr immune response

• Is integrated into the host cell genome

• Less efficient

Lentivirus

• Carries up to 8,500 base paur

• Carries RNA

• No cell specificity

• Does NOT trigger immune response

• Is integrated into the host cell genome

• efficient

Cystic Fibrosis