American researchers have successfully applied a customised CRISPR gene-editing treatment for the first time to treat a baby with carbamoyl phosphate synthetase 1 (CPS1) deficiency

In a groundbreaking medical achievement, researchers in the United States have, for the first time, effectively applied a tailored CRISPR-based gene-editing treatment to address a rare genetic condition known as Carbamoyl Phosphate Synthetase 1 (CPS1) deficiency in a baby.

CPS-1 Deficiency is an uncommon genetic metabolic condition in which the liver is unable to produce an enzyme that transforms harmful ammonia into urea, typically eliminated via urine.

What is Gene Editing Treatment?

Definition:

Gene editing therapy involves the intentional modification of DNA sequences in a person's cells to address or heal genetic disorders.

It entails altering, removing, or adding particular genes at precise sites in the genome to rectify mutations or improve cellular functions.

Categories of Gene Editing Methods:

CRISPR-Cas9: The most popular tool; it precisely cuts DNA at designated locations with the help of guide RNA and the Cas9 enzyme.

Zinc Finger Nucleases (ZFNs): Employs modified proteins to attach to and cleave DNA.

Base Editing: Modifies one nucleotide without disrupting DNA strands

Prime Editing: Functions as a 'text editor' to insert, remove, or substitute DNA sequences.

Uses of Gene Editing Therapy

Medicine: Addressing genetic conditions such as Sickle Cell Anemia, Beta-Thalassemia, CPS-1 Deficiency.

Modifying immune cells (CAR-T therapy) to attack cancer.

Agriculture: Creating high-yield, disease-resistant plants

Veterinary Science: Improving characteristics of livestock

India's Advancement in Gene Editing

CSIR-Institute of Genomics and Integrative Biology (IGIB) is creating a native CRISPR framework (IndiCRISPR).

The National Biopharma Mission is financing genome-editing research through the Department of Biotechnology (DBT).

Gene Therapy Guidelines (2020): Released to facilitate ethical clinical implementation.

Difficulties

Safety concerns: Unintended consequences, immune responses, accidental mutations.

Ethical issues: Germline modification (genetic changes passed on), custom-made infants

Restricted access: Expensive, access confined to developed countries