1st Personalised Gene-Editing Therapy

Context (IE): US doctors and researchers have successfully tested a gene-editing therapy to treat an infant diagnosed with a carbamoyl phosphate synthetase 1 (CPS-1) deficiency.
Researchers developed a bespoke CRISPR-based therapy explicitly tailored for that infant.
Unlike previous CRISPR treatments that require editing stem cells outside the body, this therapy was delivered directly into infants’ livers using lipid nanoparticles carrying RNA and a guide sequence targeting the mutation.

What is Gene Editing?

Gene editing is a cutting-edge technique that allows scientists to make precise, targeted changes to the DNA of living organisms, including plants, bacteria, and animals.
It enables adding, removing, or altering specific genetic material at particular locations in the genome.
Tools: The most popular tool is CRISPR-Cas9, a system borrowed from bacteria that acts like molecular scissors to cut DNA at a specific spot.
- Other gene editing methods include TALENs and Zinc Finger Nucleases (ZFNs).

Somatic Gene Editing	Germline Gene Editing
Somatic Gene Editing involves altering the DNA of non-reproductive (somatic) cells such as those in the skin, liver, or blood. It targets specific tissues or organs within an individual to treat diseases or modify traits. For example, gene therapy is used for sickle cell anaemia. Genetic changes are confined to the treated individual only, cannot be passed on to offspring.	Germline Gene Editing modifies the DNA in reproductive cells (sperm/eggs) or early-stage embryos. As a result, the genetic changes are present in every organism’s cell, including somatic and reproductive cells. These alterations are heritable and can be transmitted to future generations, permanently altering the genetic lineage.

Medical Treatments: Correcting genetic mutations to cure or manage diseases such as cystic fibrosis, sickle cell anaemia, muscular dystrophy, and certain cancers.
Personalised Medicine: Designing therapies tailored to an individual’s genetic makeup for more effective results.
Agriculture: Developing crops with improved yield, pest resistance, and climate resilience.
Prevention of Genetic Disorders: Particularly via germline editing to stop hereditary diseases pre-birth.

Safety Risks: Off-target mutations may cause unintended genetic changes, leading to harmful effects.
Ethical Issues: Especially with germline editing, altering human heredity raises moral questions about consent, identity, and “designer babies”.
Long-Term Impact: Unknown consequences on future generations and ecological balance.
Access and Equity: Potential for widening social inequality if gene editing is only available to affluent populations.

Strengthen Research and Development: Invest in improving the precision, efficiency, and safety of gene editing technologies like CRISPR, base editing, and prime editing.
Establish Robust Regulatory Frameworks: Create clear, globally harmonised regulations that ensure ethical use, safety, and equitable access to gene editing therapies.
Prioritise Ethical Considerations: Implement strict oversight to prevent unethical applications, such as ‘designer babies’ or genetic enhancements beyond therapeutic purposes.
Focus on Accessibility and Equity: Ensure that breakthroughs in gene editing benefit all sections of society, especially marginalised and low-income groups.

It is a rare genetic metabolic disorder that affects the urea cycle, the body’s primary mechanism for removing excess nitrogen (ammonia) produced during protein metabolism.
- Urea Cycle: Normally, our body converts excess ammonia to urea and removes it from the body through urination. CPS-1 enzyme, located in the mitochondria of liver cells, catalyses the 1^st step of the urea cycle.