Gene Therapy Delivery Vector Crosses the Blood-Brain Barrier
Source: American Journal of Medical Genetics, Part A
Clinical genome sequencing in patients with suspected rare genetic disease in Peru
Source: npj Genomic Medicine
Parkinson’s families project: a UK-wide study of early onset and familial Parkinson’s disease
Source: npj Parkinson's Disease
Different diseases, different needs: Patient preferences for gene therapy in lysosomal storage disorders, a probabilistic threshold technique survey
Source: Orphanet Journal of Rare Diseases
Therapeutic delivery of recombinant glucocerebrosidase enzyme-containing extracellular vesicles to human cells from Gaucher disease patients
Source: Orphanet Journal of Rare Diseases
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