Method of treating lbsl by enhancing dars2 expression
AssigneesThe Johns Hopkins University, Kennedy Krieger Institute, Inc., University of Maryland, Baltimore
InventorsSeyed Ali Fatemi, Christina L. Nemeth Mertz, Adam Ratajczak, Piotr Walczak, Miroslaw Janowski, Anna Jablonska, Yajie Kevin Liang
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Abstract
Provided herein are methods of treating leukoencephalopathy with brainstem and spinal cord involvement and lactate elevation (LBSL) in a subject in need thereof, the method comprising: administering an adeno-associated virus (AAV) vector to the subject, wherein the adeno-associated vector increases expression of a DARS2 gene, thereby treating LBSL.
Claims12
A method of treating leukoencephalopathy with brainstem and spinal cord involvement and lactate elevation (LBSL) in a subject in need thereof, the method comprising:
- administering an adeno-associated virus (AAV) vector to the subject, wherein the adeno-associated vector increases expression of a DARS2 gene, thereby treating LBSL.
Dependencies are inferred from the claim text.