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Gene Editing - Part III: The History of Gene Editing

Gene Editing - Part III: The History of Gene Editing

Anna Eisenberg
March 22, 2025

Anna's Deep Dives

_{Just facts, you think for yourself}

The Discovery of DNA & the Genetic Code

Scientists have studied heredity for over a century. In 1866, Gregor Mendel discovered that traits pass from one generation to the next. His work laid the foundation for genetics.

In 1869, Johann Friedrich Miescher isolated a substance from white blood cells, later identified as DNA. By the early 1900s, researchers confirmed chromosomes carry genetic information. In 1928, Frederick Griffith’s experiment hinted that DNA controls inheritance.

In 1944, Oswald Avery, Colin MacLeod, and Maclyn McCarty proved DNA, not proteins, carried genetic instructions. In 1953, James Watson and Francis Crick, using Rosalind Franklin’s X-ray images, discovered DNA’s double-helix structure, explaining how genetic information is stored and copied.

In the 1960s, scientists cracked the genetic code. Marshall Nirenberg and others identified codons, the three-letter sequences that instruct cells to build proteins. By 1966, all 64 codons were mapped, showing how DNA translates into traits.

In 1983, Kary Mullis invented polymerase chain reaction (PCR), enabling rapid DNA replication. The Human Genome Project, completed in 2003, mapped the entire human genome, identifying about 20,000 genes and three billion base pairs. This paved the way for gene editing and personalized medicine.

The Rise of Genetic Engineering: From GMOs to Gene Therapy

Genetic engineering began in 1973 when Herbert Boyer and Stanley Cohen transferred DNA between bacterial cells. This breakthrough led to genetically modified organisms (GMOs).

In 1983, scientists created the first genetically modified plant, making it resistant to herbicides. By the 1990s, GM crops like corn and soybeans became widely used. Today, over 190 million hectares of farmland grow GMOs.

Genetic engineering revolutionized medicine. In 1982, genetically modified bacteria produced human insulin, replacing animal-derived insulin for diabetes treatment. The 1990s introduced gene therapy, with the first trial treating severe combined immunodeficiency (SCID) by inserting a healthy gene.

In 2017, the FDA approved Kymriah, the first gene therapy for cancer, modifying immune cells to attack leukemia. Other therapies, like Zolgensma, treat genetic disorders such as spinal muscular atrophy. By 2023, 68.5% of gene therapy trials focused on cancer. High costs raise concerns about accessibility.

CRISPR, discovered in 2012, made gene editing faster and cheaper. Scientists now use it to modify crops, treat genetic diseases, and develop therapies. The genetic engineering market is projected to reach $50.6 billion by 2031.

CRISPR’s Breakthrough: A Nobel Prize-Winning Revolution

CRISPR-Cas9 uses a guide RNA to direct the Cas9 enzyme to a specific DNA sequence, where it makes a cut, allowing genes to be modified or replaced. This method surpasses earlier gene-editing techniques like Zinc Finger Nucleases (ZFNs) and TALENs.

CRISPR transformed genetic research. Scientists used it to edit human cells, create disease-resistant crops, and study genetic diseases. It simplified gene editing, making it widely accessible.

In 2016, the first clinical trials tested CRISPR in humans. By 2023, the FDA approved Casgevy, the first CRISPR-based therapy for sickle cell disease. The CRISPR market, valued at $9.88 billion in 2024, is expected to reach $40.48 billion by 2033, with North America holding 49% of the market share.

In 2020, Doudna and Charpentier won the Nobel Prize in Chemistry for their work on CRISPR, the first all-female team to receive a Nobel Prize in science. Their discovery opened new possibilities for treating genetic disorders, developing biofuels, and improving agriculture.

Despite its potential, CRISPR raises ethical concerns. The 2018 birth of gene-edited twins in China sparked global controversy over the risks of human germline editing. Many fear unintended genetic consequences or misuse.

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