Patent classifications
C12Y305/01015
Metabolic therapy for oxidative stress in the brain through targeted neuronal catabolism of N-acetyl-aspartic acid
The present invention provides a novel method of treatment for treating brain disorders that manifest oxidative stress by providing targeted populations of neurons with the ability to catabolize the acetylated amino acid derivative, N-acetylaspatic acid (NAA) and further supply extraphysiological levels of ATP to neurons via the targeted expression of the NAA catabolic enzyme aspartoacylase (ASPA) in neurons and astrocytes.
Methods and compositions for treating metabolic imbalance
In some aspects, the disclosure relates to compositions and methods useful for the diagnosis and treatment of diseases associated with a metabolic imbalance in a subject (e.g., cancer). In some embodiments, the methods comprise administering to a subject an N-acetylaspartate (NAA)-depleting agent or an N-acetylaspartate (NAA)-increasing agent based upon the subject's metabolic profile.
SELECTIVE RECOVERY
Provided herein are methods of selective screening. In addition, various targeting proteins and sequences, as well as methods of their use, are also provided.
Selective recovery
Provided herein are methods of selective screening. In addition, various targeting proteins and sequences, as well as methods of their use, are also provided.
Methods of Treating or Preventing Amyotrophic Lateral Sclerosis
This disclosure provides methods for treating, ameliorating, or reversing at least one symptom of amyotrophic lateral sclerosis (ALS) in a subject by increasing the amount of neuronal aspartate in spinal cord through administration of a therapeutically effective amount of a composition comprising a nucleic acid encoding ASPA or a functional fragment thereof.
Selective recovery
Provided herein are methods of selective screening. In addition, various targeting proteins and sequences, as well as methods of their use, are also provided.
CNS targeting AAV vectors and methods of use thereof
The invention in some aspects relates to recombinant adeno-associated viruses useful for targeting transgenes to CNS tissue, and compositions comprising the same, and methods of use thereof. In some aspects, the invention provides methods and compositions for treating CNS-related disorders.
METABOLIC THERAPY FOR OXIDATIVE STRESS IN THE BRAIN THROUGH TARGETED NEURONAL CATABOLISM OF N-ACETYL-ASPARTIC ACID
The present invention provides a novel method of treatment for treating brain disorders that manifest oxidative stress by providing targeted populations of neurons with the ability to catabolize the acetylated amino acid derivative, N-acetylaspatic acid (NAA) and further supply extraphysiological levels of ATP to neurons via the targeted expression of the NAA catabolic enzyme aspartoacylase (ASPA) in neurons and astrocytes.
ENGINEERED PARASITES FOR DELIVERING PROTEIN TO THE CENTRAL NERVOUS SYSTEM (CNS)
Provided are nucleic acid constructs, Toxoplasma comprising same, pharmaceutical compositions comprising same and methods using same for delivering a protein-of-interest to a tissue-of-interest of a subject, such as the CNS and further treating a pathology which is treatable by administration of a therapeutic polypeptide in a central nervous system of the subject.
SELECTIVE RECOVERY
Provided herein are methods of selective screening. In addition, various targeting proteins and sequences, as well as methods of their use, are also provided.