Gene Therapy

Many diseases are due to dysfunction of mutated genes. In traditional gene therapy, additional copies of therapeutic genes are delivered into transfected cells. This approach has several drawbacks (more info).  Recently, researchers are focusing on a new approach: direct repair of mutated genes.

Basic information from Human Genome Project Information

Review articles

New approach:

Genetic Repair - J. Clin. Invest., 2003.

• Targeted genetic repair: an emerging approach to genetic therapy
• Ribozyme-mediated revision of RNA and DNA
• Messenger RNA reprogramming by spliceosome-mediated RNA trans-splicing
• Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing
• The potential for gene repair via triple helix formation
• Targeted gene repair – in the arena
• Sequence-specific modification of genomic DNA by small DNA fragments

Gene Repair and Transposon-Mediated Gene Therapy - Stem Cells, 2002.

Traditional approach:

Novel molecular approaches to cystic fibrosis gene therapy - Biochem. J., 2005.

Dangerous liaisons: the role of "danger" signals in the immune response to gene therapy - Blood, 2002

Adeno-associated Virus for Cancer Gene Therapy - Cancer Research, 2001.

Development of lentiviral vectors for gene therapy for human diseases - Blood, 2000.

Gene delivery from replication-selective viruses: arming guided missiles in the war against cancer - J. Clinical Investigation, 2000.

Design of Retroviral Vectors and Helper Cells for Gene Therapy - Pharmacological Reviews, 2000.

Adeno-Associated Virus Vectors and Hematology - Blood, 1999.