Q1). Gene therapy is an experimental approach to treat the disease by inserting therapeutic genes or changing gene expression levels in the patient’s cells. Genes are nucleic acid polymers; these therapeutic genes used in gene therapy generally encode a functional gene that replaces mutated gene. Potential treatment for single gene genetic disorders such as haemophilia, muscular dystrophy, cystic fibrosis, etc. was demonstrated. Treatment of cancer, AIDS (acquired immune deficiency syndrome), Alzheimer’s disease and rheumatoid arthritis are also under active investigation. All the diseases cannot be cured by gene therapy. Some of hte good targets of the gene therapy include, 1). Diseases with known genetic defects or mutations 2). Diseases with point mutations on one or more genes 3). The possibility of delivering genes to the affected tissues 4). If the substitution of defective gene correct the disease status Solution Q1). Gene therapy is an experimental approach to treat the disease by inserting therapeutic genes or changing gene expression levels in the patient’s cells. Genes are nucleic acid polymers; these therapeutic genes used in gene therapy generally encode a functional gene that replaces mutated gene. Potential treatment for single gene genetic disorders such as haemophilia, muscular dystrophy, cystic fibrosis, etc. was demonstrated. Treatment of cancer, AIDS (acquired immune deficiency syndrome), Alzheimer’s disease and rheumatoid arthritis are also under active investigation. All the diseases cannot be cured by gene therapy. Some of hte good targets of the gene therapy include, 1). Diseases with known genetic defects or mutations 2). Diseases with point mutations on one or more genes 3). The possibility of delivering genes to the affected tissues 4). If the substitution of defective gene correct the disease status.

1. Q1). Gene therapy is an experimental approach to treat the disease by inserting therapeutic genes
or changing gene expression levels in the patient’s cells. Genes are nucleic acid polymers; these
therapeutic genes used in gene therapy generally encode a functional gene that replaces mutated
gene. Potential treatment for single gene genetic disorders such as haemophilia, muscular
dystrophy, cystic fibrosis, etc. was demonstrated. Treatment of cancer, AIDS (acquired immune
deficiency syndrome), Alzheimer’s disease and rheumatoid arthritis are also under active
investigation.
All the diseases cannot be cured by gene therapy. Some of hte good targets of the gene therapy
include,
1). Diseases with known genetic defects or mutations
2). Diseases with point mutations on one or more genes
3). The possibility of delivering genes to the affected tissues
4). If the substitution of defective gene correct the disease status
Solution
Q1). Gene therapy is an experimental approach to treat the disease by inserting therapeutic genes
or changing gene expression levels in the patient’s cells. Genes are nucleic acid polymers; these
therapeutic genes used in gene therapy generally encode a functional gene that replaces mutated
gene. Potential treatment for single gene genetic disorders such as haemophilia, muscular
dystrophy, cystic fibrosis, etc. was demonstrated. Treatment of cancer, AIDS (acquired immune
deficiency syndrome), Alzheimer’s disease and rheumatoid arthritis are also under active
investigation.
All the diseases cannot be cured by gene therapy. Some of hte good targets of the gene therapy
include,
1). Diseases with known genetic defects or mutations
2). Diseases with point mutations on one or more genes
3). The possibility of delivering genes to the affected tissues
4). If the substitution of defective gene correct the disease status