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Gene transfer and gene therapy,

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Contents: Hemophilia treatment past and present

Gene transfer for genetic disease

Viruses engineered to deliver genes

Gene therapy using integrating vectors

Adeno-associated virus (AAV) structure and composition

AAV-Factor IX vectors

AAV-mediated gene transfer for hemophilia

Risk of germline transmission

Peptide library based on AAV and Factor IX

Transaminitis and liver enzymes

Neutralizing antibodies to AAV

Self-complementary DNA and Factor IX synthesis

Strategies to overcome immunity to AAV

Use of empty capsids.

Genetic vectors > Therapeutic use.

Hemophilia > Gene therapy.

Hemophilia > genetics.

Hemophilia > therapy.

Dependovirus - genetics.

Gene Transfer Techniques.

Genetic Therapy - methods.

Hemophilia B - genetics.

Hemophilia B - therapy.