Non-viral delivery of CRISPR/Cas cargo to the retina using nanoparticles: Current possibilities, challenges, and limitations
The discovery of the CRISPR/Cas system and its development into a powerful genome
engineering tool have revolutionized the field of molecular biology and generated
excitement for its potential to treat a wide range of human diseases. As a gene therapy
target, the retina offers many advantages over other tissues because of its surgical
accessibility and relative immunity privilege due to its blood–retinal barrier. These features
explain the large advances made in ocular gene therapy over the past decade, including the …
engineering tool have revolutionized the field of molecular biology and generated
excitement for its potential to treat a wide range of human diseases. As a gene therapy
target, the retina offers many advantages over other tissues because of its surgical
accessibility and relative immunity privilege due to its blood–retinal barrier. These features
explain the large advances made in ocular gene therapy over the past decade, including the …
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