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Scientists saw the following changes after a 26-week treatment plan

PubMed laboratory research record [PMID: 22204800] PubMed record related to this compound for laboratory literature review

Future research should focus on vector development for enhanced specificity and safety and optimize CRISPR-based approaches for long-term gene editing in DN-related genes

Among surviving patients, the most severely affected patients have intellectual disability, with IQs ranging from the low 50s to around 60, progressive cerebellar disease and ataxia, recurrent bacterial infections, chronic metabolic acidosis, and hemolytic anemia