PRCN 323 / Neuralgene - LARVOL DELTA

From gene to therapy: a review of deciphering the role of ABCD1 in combating X-Linked adrenoleukodystrophy. (PubMed, Lipids Health Dis) - "Current therapeutic approaches are evaluated, discussing their limitations, and emphasizing the need to fully elucidate the pathogenesis of X-ALD. Additionally, this review highlights the importance of international collaboration to enhance systematic data collection and advance biomarker discovery, ultimately improving patient outcomes with X-ALD."

Journal • Review • Endocrine Disorders • Gene Therapies • Genetic Disorders • Nephrology • Renal Disease

Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model. (PubMed, Gene Ther) - "ABCD1 expression was detected 15 days later, and the impaired motor ability was gradually alleviated. Our studies established an early onset ALD model and illustrated neurological improvement after LV.ABCD1 intracerebral injection without immunopathological toxicity."

Journal • Preclinical • Gene Therapies • Genetic Disorders