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Genome editing for curing sickle-cell anemia, HIV, and cystic fibrosis and other diseases and then to create designer babies
Technology review reports on genome surgery
CRISPR is a new technology that could allow researchers to perform microsurgery on genes, precisely and easily changing a DNA sequence at exact locations on a chromosome. CRISPR changed everything. It replaces the DNA-targeting proteins with a short bit of RNA that homes in on desired genes. Unlike the complex proteins, RNA—which has nearly the same simple structure as DNA—can be made routinely in the lab; a technician can quickly synthesize the roughly 20-letter-long sequences the method requires. The system makes it easy for medical researchers to modify a genome by replacing, deleting, or adding DNA.
CRISPR stands for “clustered regularly interspaced short palindromic repeats”—clusters of brief DNA sequences that read similarly forward and backward, which are found in many types of bacteria. Scientists first observed the puzzling DNA segments in the 1980s but didn’t understand for almost two decades that they are part of a bacterial defense system. When a virus attacks, bacteria can incorporate sequences of viral DNA into their own genetic material, sandwiching them between the repetitive segments. The next time the bacteria encounter that virus, they use the DNA in these clusters to make RNAs that recognize the matching viral sequences. A protein attached to one of these RNAs then cuts up the viral DNA.
It will be several years before CRISPR can be developed into human therapeutics, but a growing number of academic researchers have seen some preliminary success with experiments involving sickle-cell anemia, HIV, and cystic fibrosis.
Source: http://nextbigfuture.com/2014/02/genome-editing-for-curing-sickle-cell.html
