Genetic Disorders and Gene Therapy

Genome editing with engineered nucleases (GEEN) is emergent type of Genetic Engineering. GEEN is the technology in which DNA is inserted, deleted or replaced in the genome. The emergence of highly versatile genome-editing technologies has provided investigators with the ability to rapidly and economically introduce sequence-specific modifications into the genomes of a broad spectrum of cell types and organisms. It also promotes various changes in sub cellular level. Genome Editing itself also holds tremendous potential for treating the underlying various idiopathic genetic causes of certain diseases. The core technologies now most commonly used techniques to facilitate genome editing are clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription activator-like effector nucleases (TALENs), zinc-finger nucleases (ZFNs), and homing endonucleases or mega nucleases.

• Stem cell and cellular therapies
• Cell therapy: clinical trials
• Paediatrics’ and Genetics
• Market and Future Prospects of Gene Therapy
• Gene Editing and CRISPR Based Technologies
• meganucleases
• Gene targeting