Genetic technology applied to medicine — common mistakes

Remember to differentiate between gene therapy (introducing whole new genes, often to supplement) and gene editing (precisely altering existing genes). CRISPR-Cas9 is a powerful gene editing tool, allowing for corrections rather than just additions.

In Cambridge Biology 9700, genetic technology applied to medicine encompasses the use of advanced techniques to understand, diagnose, and treat genetic conditions. This includes genetic screening (identifying predispositions or diseases), gene therapy (introducing functional genes to correct disorders), and sophisticated gene editing tools like CRISPR-Cas9 (making precise changes to DNA). Mastering these concepts is vital for Paper 4, and you can practise with our free course materials and past papers.

Gene therapy aims to treat genetic diseases by delivering healthy copies of genes into a patient's cells, either ex vivo (outside the body) or in vivo (inside the body). This is typically achieved using vectors like modified viruses or liposomes that carry the therapeutic DNA. Key limitations include the challenge of targeted and efficient gene delivery, potential immune responses to vectors, the risk of insertional mutagenesis, achieving long-term gene expression, and the high cost, which can limit accessibility.

CRISPR-Cas9 and genetic screening raise significant ethical dilemmas. For screening, concerns include privacy of genetic data, potential for discrimination, and the moral status of embryos in PGD. For CRISPR-Cas9, beyond safety and off-target effects, there's the debate over germline editing (changes passed to future generations) versus somatic cell editing, and the 'slippery slope' argument regarding its potential use for enhancement rather than just disease treatment, leading to 'designer babies' and exacerbating social inequalities. Our free revision resources delve deeper into these crucial discussions.