New Gene Editing Method: Future of Personalized Medicine Explained

A new gene editing method promises breakthroughs in personalized medicine by enabling precise treatment of genetic disorders, improving therapy effectiveness, and reducing side effects, while raising ethical and regulatory challenges.

Table of Contents

Context

Researchers recently developed a treatment called PERT (Prime-Editing-mediated Readthrough of premature Termination codons) for the nonsense mutation .

More about PERT

Instead of fixing every mutation separately, it reprogram one of the cell’s own tRNA genes to ignore premature stop codons. This makes the therapy gene-agnostic (works across multiple diseases).

Protein Synthesis in the Body

1. DNA → RNA → Protein

● DNA contains genetic instructions.

● DNA is copied into mRNA (messenger RNA).

● mRNA is read in sets of three letters called codons.

● Each codon codes for one amino acid (building block of proteins).

● Ribosomes assemble amino acids into proteins.

2. Role of tRNA

● tRNA (transfer RNA) acts like a translator.

● It reads codons and brings the correct amino acid.

● Stop codons signal the ribosome to end protein production.

3. Problem in Nonsense Mutation

● A nonsense mutation inserts a premature stop signal (stop codon) into a gene. This causes protein production to stop midway, producing an incomplete and non-functional protein.

● Around 25% of disease-causing mutations are nonsense mutations. These are seen in diseases like:

○ Cystic fibrosis

○ Tay-Sachs disease

○ Batten disease

○ Niemann-Pick disease

○ Hurler syndrome

How PERT Works for Nonsense Mutations

It is a precise genome-editing tool (advanced form of CRISPR) that can rewrite DNA without cutting both strands.

It uses:

1. pegRNA (Prime-editing guide RNA)

● Guides the editing machinery to the exact DNA location.

● Carries template for new genetic sequence.

2. Prime Editing Enzyme (PE6c)

● Special enzyme identified in this study.

● Rewrites the targeted tRNA gene efficiently.

3. PE3 Strategy

● Uses an additional guide RNA.

● Increases chances that the cell adopts the edited sequence.

Difference between PERT and CRISPR based editing

● CRISPR-Based Editing edits the faulty DNA directly. However it is often mutation-specific and complex. Thus it is disease-specific, expensive, and time-consuming.

Why This Strategy is Important

One Strategy for Many Diseases: Since ~25% of genetic disorders involve nonsense mutations, PERT could treat multiple diseases.
Gene-Agnostic Therapy: Does not target a specific gene — targets the mutation type.
High Editing Efficiency: Much higher than many traditional gene-editing methods
- g. PERTT has 60–80% success rate in human cells. It is much higher than homology-directed repair (10–20%)..

Clinical Possibility: Recent successful base-editing therapy in humans suggests delivery systems are feasible.

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