Gene Therapy

Gene Therapy involves altering genes in the body's cells to treat disease through the production of good protein transcripts or the use of siRNA.

Transfection is the introduction of nucleic acids into animal cells, while transduction refers to virus-mediated DNA transfer.

Studying protein expression in eukaryotic cells allows for proper folding and post-translational modifications.

RNA transfection allows for knock-down and knock-in experiments.

Transfection targets can include DNA, RNA, and protein.

Transient and stable transfection options are available.

Transient transfection does not integrate DNA into the genome and produces a high level of expression for a few days, while stable transfection results in integration and long-term expression.

Transfection methods include cationic lipid, electroporation, in vivo, co-transfection, RNAi/siRNA, transient, stable, calcium phosphate, CRISPR, WHDNA, and mRNA transfection.

Cloning vectors, including plasmids, cosmids, and phages, are small pieces of DNA employed to introduce foreign genes of interest into the host cell.

Expression vectors are employed to express a foreign gene, consisting of a promoter to drive target gene expression, plasmid, and antibiotic resistance gene.

Electroporation is a physical method of DNA transfection with high cell death and low efficiency but prevents DNA degradation.

Calcium phosphate transfection is a buffered solution mixed with Plasmid DNA and adheres to the cell surface - a cheap method with high efficiency.

Gene Therapy involves altering genes in the body's cells to treat disease through the production of good protein transcripts or the use of siRNA.

Transfection is the introduction of nucleic acids into animal cells, while transduction refers to virus-mediated DNA transfer.

Studying protein expression in eukaryotic cells allows for proper folding and post-translational modifications, while RNA transfection allows for knock-down and knock-in experiments.

Transfection targets can include DNA, RNA, and protein, with transient and stable transfection options available.

Transient transfection does not integrate DNA into the genome and produces a high level of expression for a few days, while stable transfection results in integration and long-term expression.

Transfection methods include cationic lipid, electroporation, in vivo, co-transfection, RNAi/siRNA, transient, stable, calcium phosphate, CRISPR, WHDNA, and mRNA transfection.

Cloning vectors, including plasmids, cosmids, and phages, are small pieces of DNA employed to introduce foreign genes of interest into the host cell.

Expression vectors are employed to express a foreign gene, consisting of a promoter to drive target gene expression, plasmid, and antibiotic resistance gene.

Electroporation is a physical method of DNA transfection with high cell death and low efficiency but prevents DNA degradation.

Calcium phosphate transfection is a buffered solution mixed with Plasmid DNA and adheres to the cell surface - a cheap method with high efficiency.

The use of viral vectors, specifically retroviruses, adenoviruses, lentiviruses, and adeno-associated viruses, can infect and insert genetic material into both dividing and non-dividing cells.

Gene therapy treatment for Severe Combined Immunodeficiency (SCID) involves removing blood stem cells and transplanting the working copy of the ADA gene via viral transduction, but it can lead to cancer (25%).