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CRISPR-Cas9






๐Ÿ“Œ Topic 03 of 6 ยท Chapter 04 ยท Biotechnology & Genetic Engineering

CRISPR-Cas9 Gene Editing Technology

How CRISPR works, applications, Nobel Prize 2020, ethical concerns โ€” complete notes for UPSC & PSC exams.

โœ‚๏ธ CRISPR-Cas9 โ€” What is it?

  • CRISPR = Clustered Regularly Interspaced Short Palindromic Repeats
  • Cas9 = CRISPR-associated protein 9 โ€” acts as “molecular scissors” to cut DNA at specific locations
  • Originally discovered as a bacterial immune system against viruses
  • Adapted as a gene editing tool by Jennifer Doudna and Emmanuelle Charpentier (2012)
  • Nobel Prize in Chemistry 2020 โ€” Jennifer Doudna (USA) and Emmanuelle Charpentier (France)

โœ‚๏ธ How CRISPR-Cas9 Works

  • Step 1: Design a “guide RNA” (gRNA) that matches the target DNA sequence
  • Step 2: gRNA guides the Cas9 protein to the exact location in the genome
  • Step 3: Cas9 cuts both strands of DNA at the target location
  • Step 4: Cell’s natural repair mechanisms fix the cut โ€” gene can be disabled, corrected, or new gene inserted
  • Much more precise, cheaper, and faster than previous gene editing tools (TALEN, ZFN)

โœ‚๏ธ Applications of CRISPR

ApplicationDetails
Treating genetic diseasesSickle cell disease, thalassemia, Huntington’s disease โ€” first CRISPR therapy approved (2023) for sickle cell
Cancer therapyEngineering immune cells (T-cells) to attack cancer
Disease-resistant cropsDeveloping crops resistant to diseases without introducing foreign genes
Gene drivesSpreading genetic changes through wild populations โ€” e.g., eliminating malaria-carrying mosquitoes
Drug discoveryCreating disease models; identifying drug targets

โœ‚๏ธ Ethical Concerns

  • “Designer babies” โ€” editing embryo genes for non-medical traits (intelligence, appearance)
  • Germline editing โ€” changes passed to future generations; irreversible
  • Off-target effects โ€” unintended cuts in wrong DNA locations
  • He Jiankui controversy (2018) โ€” Chinese scientist edited human embryos โ†’ twin girls born with edited CCR5 gene โ†’ global outrage; sentenced to prison
  • Most countries ban germline editing for reproductive purposes
๐Ÿ“Œ Key Exam Points:
โ€ข CRISPR = Clustered Regularly Interspaced Short Palindromic Repeats
โ€ข Cas9 = “molecular scissors” = cuts DNA at specific location
โ€ข Nobel Prize Chemistry 2020 = Jennifer Doudna + Emmanuelle Charpentier
โ€ข First CRISPR therapy approved (2023) = sickle cell disease
โ€ข He Jiankui (2018) = first CRISPR babies = global controversy