RNA EDITING AND HEALTH CARE, PM- Vidyalaxmi Scheme, RNA Editing

(General Studies III – Science & Technology section – Science and Technology- Developments and their Applications and Effects in Everyday Life.)

• RNA editing, a transformative tool in genetic medicine, enables temporary corrections to RNA sequences, offering an alternative to permanent DNA editing.
• With the potential to treat genetic disorders safely and reversibly, RNA editing marks a new era in precision medicine.

Recent advancements in RNA editing showcase its potential in treating severe conditions. Wave Life Sciences achieved a breakthrough by correcting single-point mutations in the SERPINA1 gene to treat alpha-1 antitrypsin deficiency (AATD), normalizing protein levels. Companies like Korro Bio and ProQR Therapeutics are developing RNA-based therapies for Parkinson’s and cardiovascular diseases.

Potential Applications –

1. Neurological Disorders: Targeting mRNA transcripts associated with conditions like Parkinson’s disease and amyotrophic lateral sclerosis (ALS) to correct pathogenic mutations.
2. Cardiovascular Diseases: Modifying RNA to address genetic mutations leading to heart conditions, potentially offering treatments for inherited cardiomyopathies.
3. Oncology: Editing RNA in cancer cells to correct aberrant splicing events or mutations, thereby inhibiting tumor growth and progression.
4. Metabolic Disorders: Correcting RNA sequences in metabolic pathways to treat conditions like phenylketonuria or familial hypercholesterolemia.
5. Ophthalmic Diseases: Addressing genetic mutations in retinal cells to treat inherited eye disorders such as retinitis pigmentosa.

Limitations –

1. Off-Target Effects: Unintended edits can occur, leading to unforeseen consequences.
2. Delivery Challenges: Efficiently delivering RNA editing components to specific tissues remains a significant hurdle.
3. Transient Effects: RNA edits are temporary, necessitating repeated treatments to maintain therapeutic benefits.
4. Immunogenicity: The introduction of exogenous RNA or proteins can trigger immune responses, complicating therapy.
5. Ethical and Regulatory Concerns: The long-term effects and ethical implications of RNA editing require thorough evaluation before widespread clinical adoption.

Future Prospects –

• The RNA editing market is still in its infancy, with at least 11 companies actively pursuing this technology. Major pharmaceutical players like Eli Lilly and Roche have shown interest.
• Continued research and clinical trials promise to integrate RNA editing into mainstream medical practice, addressing a range of genetic and acquired conditions.

• Vehicles in Delhi contributed more to air pollution than stubble burning between October 12 and November 3, according to the Centre for Science and Environment (CSE)’s Mobility crisis is behind the pollution in Delhi Report.
• Out of total pollution sources, neighbouring districts contributed 34.97%, Delhi’s local sources 30.34% and farm fires 8.19%.

• Among Delhi’s local sources, vehicular emissions accounted for 51.5%, with dust contributing 3.7%.
• The findings are based on data from the Decision Support System for Air Quality Management in Delhi, developed by the Indian Institute of Tropical Meteorology (IITM), Pune.
• Cutting emissions from all sources is critical, with an immediate focus on addressing vehicular pollution to manage the mobility crisis.

Dig Deeper: Read about the impact of PM 2.5 pollution on human health. 

• The PM Vidyalaxmi Scheme is a new Central Sector scheme that seeks to provide financial support to meritorious students pursuing higher studies.
• Eligibility: Students enrolled in recognized Quality Higher Educational Institutions (QHEIs) in India are eligible covering 860 qualifying QHEIs.
• No Guarantor or Collateral Required: The scheme offers collateral-free loans, making it accessible to a wider range of students. Loan amounts up to Rs.7.5 lakhs will be provided a 75% credit guarantee by the Government of India.
• Interest Subsidy: The government provides interest subsidies for specific categories of students, including those from low-income families. 3% interest subvention for loans up to Rs.10 lakhs during the moratorium period for eligible students.
• The loan can be used to cover tuition fees, hostel fees, and other educational expenses.
• The loan repayment period is typically 5-15 years, depending on the course duration and loan amount.
• It will be complementary to the Pradhan Mantri Uchchatar Shiksha Protsahan (PM-USP) Scheme.

Dig Deeper: Read about National Education Policy, 2020

• Recently, a biotechnology company Wave Life Sciences in the USA became the first company to treat a genetic condition by editing RNA at the clinical level.
• RNA editing fixes errors in messenger RNA (mRNA) during transcription, preventing the production of faulty proteins responsible for genetic disorders.

• It utilizes adenosine deaminase enzymes (ADAR) paired with guide RNA (gRNA) to correct mutations in mRNA.
• Wave Life Sciences treated alpha-1 antitrypsin deficiency (AATD) using RNA editing to restore normal protein levels.
• It is also developing RNA editing for Huntington’s disease, Duchenne muscular dystrophy, and obesity.
• RNA editing is advantageous over DNA editing as temporary changes reduce long-term risks compared to permanent genome alterations in DNA editing.
• Use of naturally occurring ADAR enzymes minimizes immune reactions.
• Challenges in RNA Editing:
• Non-targeted editing by ADAR can lead to side effects.
• Repeated treatments are needed to sustain therapeutic effects.
• Lipid nanoparticles and viral vectors have restricted carrying capacities.

Dig Deeper: Read about RNA interference.