We report a genome-editing strategy to correct compound heterozygous mutations, a common genotype in patients with recessive genetic disorders. Adeno-associated viral vector delivery of Cas9 and guide RNA induces allelic exchange and rescues the disease phenotype in mouse models of hereditary tyrosinemia type I and mucopolysaccharidosis type I. This approach recombines non-mutated genetic information present in two heterozygous alleles into one functional allele without using donor DNA templates.
Pharmacology, Toxicology and Pharmaceutical Science
2
2%
Other
5
5%
Unknown
34
33%
Attention Score in Context
Attention Score in Context
This research output has an Altmetric Attention Score of 39. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 26 May 2026.
All research outputs
#1,241,761
of 29,792,310 outputs
Outputs from Nature Biotechnology
#2,151
of 8,843 outputs
Outputs of similar age
#19,689
of 346,398 outputs
Outputs of similar age from Nature Biotechnology
#35
of 71 outputs
Altmetric has tracked 29,792,310 research outputs across all sources so far. Compared to these this one has done particularly well and is in the 95th percentile: it's in the top 5% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 8,843 research outputs from this source. They typically receive a lot more attention than average, with a mean Attention Score of 46.0. This one has done well, scoring higher than 75% of its peers.
Older research outputs will score higher simply because they've had more time to accumulate mentions. To account for age we can compare this Altmetric Attention Score to the 346,398 tracked outputs that were published within six weeks on either side of this one in any source. This one has done particularly well, scoring higher than 94% of its contemporaries.
We're also able to compare this research output to 71 others from the same source and published within six weeks on either side of this one. This one has gotten more attention than average, scoring higher than 50% of its contemporaries.
