New Type of Technique may boost the Gene Editing Market as it can Replace or Install Gene-Sized DNA Sequence
Posted On April 16, 2022
Prime editing was introduced to the world in the year 2019. It refers to a precise approach for performing a wide range of gene alterations within human cells, including minor substitutions, insertions, and deletions. Now, advancing the practice further, researchers have created a new type of primary editing that can replace or install gene-sized DNA sequences. Twin prime editing, a CRISPR-based gene-editing technology, has enormous potential within Gene Editing Market as it could provide a novel and safer route to gene therapy.
The researchers used twinPE to modify a gene linked to Hunter syndrome (a rare genetic condition in human cells), demonstrating its therapeutic potential. An inversion of a specific forty thousand base pair long segment of DNA causes this disorder. The researchers utilized twinPE to introduce a similar-length inversion in the same place in the genome, demonstrating how the approach could fix the disease-causing mutation. The researchers also used twinPE to precisely insert thousands of base pairs of gene-sized DNA cargo inside therapeutically relevant sites present in the genome.
The method helps circumvent a flaw in the original prime editing system. In the prior processes, gene-editing could only edit a few dozen base pairs. However, some genetic illnesses may necessitate more extensive editing to investigate or treat them. TwinPE, like the original prime editing method, does not entirely split the DNA double helix by cutting both strands simultaneously, resulting in inconsistent editing results and severe chromosomal abnormalities.
A prime editor protein and two prime editing guide RNAs have been incorporated into the system. They act as guides, editing the machinery and encoding edits. Each of the two guide RNAs instructs the editing protein to form a single-stranded nick in the DNA at distinct targeted spots throughout the genome. The process successfully avoids the type of double-strand break that can result in undesirable byproducts in other approaches.
The team describes twin prime editing (twinPE), which uses two neighboring prime edits to insert more extensive DNA sequences at targeted sites in the genome while minimizing undesirable consequences. TwinPE could be a safer and more precise technique for inserting complete genes of therapeutic interest into designated positions. This may involve the native gene's location in healthy people or 'safe harbour' areas known to reduce the likelihood of side effects.
With further refinement, the technology could be employed as novel gene therapy to introduce therapeutic genes into defective or missing genes in a safe and highly targeted manner.