Tag: Gene Therapy

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Genetic editing of human embryos in the United States ignites debate

In December 2018, a Chinese researcher, He Jiankui, shocked the world when he revealed the birth of the world’s first genetically edited babies. While it is clear that Jiankui egregiously violated university regulations and ethical standards, his announcement has since ignited a heated international dialogue about the permissibility of human embryonic gene editing. Currently, there are scientists in the United States working in university laboratories, … Continue reading Genetic editing of human embryos in the United States ignites debate

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Dogs with muscular dystrophy receive CRISPR treatment: What does this mean for humans?

In an exciting step forward, dogs with Duchenne muscular dystrophy (DMD) were treated with the CRISPR genome editor to restore production of the missing protein responsible for the disease. After more studies to prove it is safe and effective long-term, this has the potential to move into clinical trials as a DMD therapy. “[The dogs] showed obvious signs of behavioral improvement – running, jumping – … Continue reading Dogs with muscular dystrophy receive CRISPR treatment: What does this mean for humans?

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A major obstacle to CRISPR/Cas9 – preexisting immunity

The discovery of CRISPR/Cas9 is one of the biggest scientific breakthroughs in recent memory. This technology, first discovered in microbes, enables the cleaving of DNA in order to remove or replace existing genes. For a crash course into the history of CRISPR/Cas9 discovery and development with primary sources please refer to the footnote*. This technology will enable us cure many genetic diseases. Work is currently … Continue reading A major obstacle to CRISPR/Cas9 – preexisting immunity

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Viruses, not all are bad for you

When you think of viruses, the yearly flu or even the Ebola or Swine flu outbreaks may come to mind. However, not all viruses cause disease – some even provide cures! Adeno-associated virus (AAV) can infect humans, but is not known to cause disease. In other words, this virus is good at getting its genetic information (genes) into human cells. What if its genes were … Continue reading Viruses, not all are bad for you

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CRISPR 2.0: Genome engineering made easy as A-B-C

CRISPR 2.0 is causing quite the ruckus in the scientific community. Why? Imagine that you had written a note in permanent marker, but later decided you wanted to change a single word. Without the ability to erase, your options would be limited, and further changes might make the note illegible. New CRISPR technologies, or “base editors,” behave as molecular erasers. These molecular erasers enable you to very precisely … Continue reading CRISPR 2.0: Genome engineering made easy as A-B-C

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Art Gallery, Images, Special Edition on Climate Change

Masquerade

Gene therapy is an approach to treating genetic diseases by re-introducing a functional copy of a gene into cells to replace the mutated, disease-causing gene. To get these genes into the cells, scientists create a vector by packaging the DNA encoding the correct gene into the outer shell–called the capsid–of a virus. A vector retains the ability of the virus to enter human cells and … Continue reading Masquerade

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Mouse embryo
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Gene editing emerges as a new therapeutic strategy for Duchenne muscular dystrophy

Advances in gene editing technology have spurred considerable progress towards a treatment for Duchenne muscular dystrophy (DMD).  Although the disease is rare – affecting roughly 1 in 5,000 male births – its consequences are devastating: patients are confined to wheelchairs at an early age and often succumb to heart or respiratory failure in their twenties or thirties.  No treatments are currently available, but three separate … Continue reading Gene editing emerges as a new therapeutic strategy for Duchenne muscular dystrophy

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Why ‘3-parent babies?’ DNA from Mom and Dad. Healthy mitochondria from another.

Mitochondria provide the energy for cells, but unlike DNA, they are only inherited from our mothers through their eggs. Defective mitochondria cause diseases which are incurable in adulthood. However, they are treatable before conception with “cytoplasmic transfer,” where defective mitochondria are swapped out for healthy ones. Continue reading Why ‘3-parent babies?’ DNA from Mom and Dad. Healthy mitochondria from another.

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