UPSC Prelims 2026Question 41Science & Technology

Which of the following statements with regard to genetic medicine is/are correct ?
1. Genetic medicines correct/compensate for the faulty genes responsible for disease.
2. Engineered viruses and lipid nanoparticles are used as carriers of the genetic medicine.
3. Genetic medicines alter the entire DNA sequence.
Select the answer using the code given below:

Options

(a)1 only
(b)2 and 3 only
(c)1 and 2 only
(d)1, 2 and 3

Correct answer: c

Decision rule

Totalising language in biotechnology — the entire genome, all cells, permanently — overshoots; real therapies act on specific genes, mutations or transcripts.

Explanation

Correct Answer

(c) 1 and 2 only

Explanation of the Correct Answer

Statement 1: Genetic medicines correct/compensate for the faulty genes responsible for disease.

Correct.

Genetic medicine refers to treatments that use genetic material or gene-based technologies to treat disease.
They may work by:
- replacing a faulty gene,
- correcting a disease-causing mutation,
- silencing a harmful gene,
- adding a functional copy of a gene,
- modifying gene expression.
Therefore, they can correct or compensate for faulty genes responsible for disease.

Statement 2: Engineered viruses and lipid nanoparticles are used as carriers of the genetic medicine.

Correct.

Genetic medicines need a delivery vehicle to carry genetic material into target cells.
Common carriers include:
- engineered viral vectors, such as adeno-associated viruses and lentiviruses,
- lipid nanoparticles, especially for mRNA and RNA-based therapies.
Lipid nanoparticles were widely discussed during the use of mRNA vaccines, where they helped deliver mRNA into cells.

Statement 3: Genetic medicines alter the entire DNA sequence.

Incorrect.

Genetic medicines do not alter the entire DNA sequence.
They usually target specific genes, specific mutations, or specific gene-expression pathways.
Some genetic medicines may not alter DNA at all. For example:
- mRNA medicines provide temporary instructions to cells.
- siRNA therapies silence specific messenger RNA.
- gene therapy may add a working gene without changing the entire genome.
- gene editing targets specific DNA sequences, not the whole DNA sequence.

Therefore, only statements 1 and 2 are correct.

Why Other Options Are Incorrect

Option	Verdict	Explanation
(a) 1 only	Incorrect	Statement 1 is correct, but statement 2 is also correct because engineered viruses and lipid nanoparticles can be used as delivery carriers.
(b) 2 and 3 only	Incorrect	Statement 2 is correct, but statement 3 is incorrect because genetic medicines do not alter the entire DNA sequence.
(c) 1 and 2 only	Correct	Genetic medicines can correct/compensate for faulty genes, and they may use engineered viruses or lipid nanoparticles as carriers.
(d) 1, 2 and 3	Incorrect	Statement 3 is incorrect. Genetic medicines are targeted and do not alter the entire genome.

Relevant Supplementary Information

Genetic Medicines: Key Types

Type of Genetic Medicine	How It Works	Example / Use
Gene therapy	Adds, replaces, or modifies a gene to treat disease	Inherited genetic disorders
Gene editing	Precisely changes selected DNA sequences	CRISPR-based therapies
mRNA medicine	Provides temporary genetic instructions to make a protein	mRNA vaccines, protein replacement approaches
siRNA therapy	Silences specific messenger RNA to reduce harmful protein production	Certain rare genetic and metabolic diseases
Antisense oligonucleotide therapy	Binds to RNA to modify gene expression or splicing	Spinal muscular atrophy-related therapies
CAR-T cell therapy	Genetically modifies immune cells to attack cancer	Blood cancers

Delivery Systems Used in Genetic Medicine

Carrier / Delivery System	What It Carries	Important Feature
Adeno-associated virus vector	DNA/gene cassette	Commonly used in gene therapy; relatively low pathogenicity
Lentiviral vector	Genetic material for stable integration	Used in some cell therapies
Adenoviral vector	Genetic instructions	Strong immune response; used in some vaccines and therapies
Lipid nanoparticles	mRNA, siRNA, RNA molecules	Non-viral delivery system; important in mRNA vaccines
Ex vivo delivery	Modified cells outside the body	Used in CAR-T and some blood disorder therapies
In vivo delivery	Genetic medicine delivered directly into the body	Used for targeting organs such as liver, eye, or muscle

Delivery System	Type	What It Carries	Important Point
Adeno-associated virus vector	Viral vector	DNA / gene cassette	Common in gene therapy; relatively low pathogenicity
Lentiviral vector	Viral vector	Genetic material	Can integrate into host-cell genome; used in some cell therapies
Adenoviral vector	Viral vector	Genetic instructions	Can trigger strong immune response
Lipid nanoparticles	Non-viral carrier	mRNA, siRNA or other RNA molecules	Widely used in mRNA vaccines and RNA therapies
Polymer nanoparticles	Non-viral carrier	DNA or RNA molecules	Being explored for targeted delivery
Ex vivo modified cells	Cell-based delivery	Genetically modified patient or donor cells	Used in CAR-T and some gene therapies
In vivo direct delivery	Direct body delivery	Genetic material into target tissues	Used for liver, eye, muscle and blood-related targets

Genetic Medicine vs Conventional Medicine

Basis	Genetic Medicine	Conventional Medicine
Target	Gene, RNA, or genetic pathway	Protein, enzyme, receptor, pathogen, or symptom
Mechanism	Corrects, compensates, silences, or modifies genetic expression	Blocks, stimulates, replaces, or suppresses biological activity
Duration	May be long-lasting or temporary depending on technology	Usually requires repeated dosing
Examples	Gene therapy, mRNA therapy, siRNA therapy, CRISPR therapy	Antibiotics, painkillers, antihypertensives
Precision	Often highly targeted	May be broad or targeted depending on drug
Genome-wide alteration?	No	Not applicable

Key Takeaways from the Tables

Point	Explanation
Genetic medicines target faulty genes or gene expression	They may correct, replace, silence, or compensate for disease-causing genetic defects.
Vectors are important	Engineered viruses and lipid nanoparticles are common delivery systems.
They do not alter the entire DNA sequence	They are targeted therapies and may not even permanently alter DNA.
mRNA and siRNA medicines are temporary	They work mainly through RNA and do not change the genome.
Gene editing is precise	Technologies like CRISPR aim at selected DNA sequences, not the entire genome.

Point	Explanation
Genetic medicine is targeted	It acts on specific genes, RNA molecules or gene-expression pathways.
It does not rewrite the whole genome	The statement that genetic medicines alter the entire DNA sequence is incorrect.
Delivery system is crucial	Viral vectors and lipid nanoparticles help carry genetic material into cells.
RNA-based medicines are usually temporary	mRNA and siRNA therapies generally do not alter DNA.
Gene editing is precise but not risk-free	It targets selected DNA regions but may raise concerns about off-target effects.
Genetic medicine is broader than gene editing	It includes gene therapy, RNA therapy, cell therapy and other gene-based approaches.

Context and Current Relevance

Genetic medicine is transforming treatment for rare genetic diseases, cancers and some metabolic disorders.
mRNA vaccines popularized the use of lipid nanoparticles for delivering genetic instructions.
The key ethical and regulatory concerns relate to safety, off-target effects, affordability and germline editing, not alteration of the entire DNA sequence.

Table: Genetic Medicine — Key Features

Feature	Details
Meaning	Medical treatment using genes, genetic material, RNA, or gene-modifying tools
Main purpose	To correct, replace, compensate for, silence, or modify faulty genetic information
Target	Specific gene, RNA, mutation, or gene-expression pathway
Does it alter entire DNA?	No
Possible duration	Temporary or long-lasting, depending on the method
Main fields of use	Rare genetic diseases, cancers, metabolic disorders, neurological disorders, immune disorders
Important delivery systems	Engineered viruses, lipid nanoparticles, modified cells
Major concern	Safety, immune response, off-target effects, cost and ethical issues

Table: Major Types of Genetic Medicines

Type	How It Works	Does It Change DNA?	Example / Use
Gene therapy	Adds or replaces a functional gene	Sometimes, depending on method	Inherited genetic disorders
Gene editing	Makes targeted changes in selected DNA sequences	Yes, but only at specific sites	CRISPR-based therapies
mRNA medicine	Provides temporary instructions to make a protein	No	mRNA vaccines, protein replacement approaches
siRNA therapy	Silences specific messenger RNA	No	Some metabolic and rare diseases
Antisense oligonucleotide therapy	Alters RNA processing or blocks harmful RNA activity	No	Spinal muscular atrophy-related therapy
CAR-T cell therapy	Genetically modifies immune cells outside the body	Yes, in modified immune cells	Certain blood cancers
DNA vaccine	Delivers DNA instructions to produce antigen	Usually does not alter host genome	Some vaccine platforms

Table: Viral Vector vs Lipid Nanoparticle

Basis	Viral Vector	Lipid Nanoparticle
Nature	Engineered virus	Fat-like particle
Main role	Delivers genetic material into cells	Delivers RNA or other genetic material into cells
Commonly carries	DNA or gene cassette	mRNA, siRNA, RNA molecules
Duration of effect	Can be long-lasting	Usually temporary
Immune response risk	Possible immune reaction against viral vector	Possible inflammatory reaction, but generally non-viral
Genome integration	Some vectors may integrate; others usually do not	Does not integrate into genome
Example use	Gene therapy, CAR-T cell engineering	mRNA vaccines, RNA medicines

Table: Genetic Medicine vs Gene Editing vs Genetic Testing

Term	Meaning	Purpose	Example
Genetic medicine	Broad category using genetic material or gene-based mechanisms for treatment	Treat or prevent disease	Gene therapy, mRNA therapy, siRNA therapy
Gene therapy	Adds, replaces, or modifies a gene	Correct or compensate for faulty gene function	Functional gene delivery
Gene editing	Precisely changes selected DNA sequences	Correct specific mutation or disable harmful gene	CRISPR-Cas systems
Genetic testing	Analyses DNA, chromosomes or genes	Diagnose risk, carrier status or disease	BRCA testing, prenatal genetic tests
Genomics	Study of the whole genome	Understand genes and their interactions	Population genomics, disease genomics
Pharmacogenomics	Studies how genes affect drug response	Personalised medicine	Choosing drug/dose based on genetic profile

Table: Similar Prelims-Trap Facts on Genetic Medicine

Statement / Theme	Correct Understanding	Common Trap
Genetic medicines correct or compensate for faulty genes	Correct	Assuming they only replace genes
Engineered viruses are used as carriers	Correct	Thinking all viruses are harmful and cannot be used medically
Lipid nanoparticles are used as carriers	Correct	Associating them only with vaccines
Genetic medicines alter entire DNA sequence	Incorrect	They target specific genes, RNA or pathways
mRNA medicines alter DNA	Incorrect	mRNA works in cytoplasm and is temporary
Gene editing changes selected DNA sites	Correct	Thinking it rewrites the whole genome
REDD+ and genetic medicine	Unrelated concepts	Confusing “gene” with “green” or climate terms in interdisciplinary questions
Genetic testing is treatment	Incorrect	Genetic testing is diagnostic, not therapy

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