Abstract: The disclosure provides compositions and methods for the Vectored Augmentation of the Destruction, Expression and/or Regulation of proteins, e.g., VA-DER systems and methods.

Abstract: The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of GCase proteins, whether in vitro and/or in vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Parkinson Disease or related condition resulting from a deficiency in the quantity and/or function of GBA gene product or associated with decreased expression or protein levels of GCase protein.

Abstract: The disclosure relates to compositions, methods, and processes for the preparation, use, and/or formulation of adeno-associated virus (AAV) particle comprising a viral gene and a capsid, wherein the viral genome comprises at least one Vectorized Editing of Nucleic acids to correct Overt Mutations (VENOM) element.

Abstract: The present invention is a method for inhibiting or reducing disease progression in subjects suffering from conditions or diseases related to the A? peptide, including Alzheimer's disease, Down's syndrome, cerebral amyloid angiopathy, mild cognitive impairment, and the like. The method comprises admininstering an apoE2 lentiviral vector to the subject.

Abstract: The present invention is a method for effecting rapid improvement in cognition in subjects suffering from conditions or diseases related to the A? peptide, including Alzheimer's disease, Down's syndrome, cerebral amyloid angiopathy, mild cognitive impairment, and the like. The method comprises administering anti-A? antibodies to the subject, especially antibodies having a high affinity for soluble forms of A?.

Abstract: A method to treat conditions characterized by formation of amyloid plaques both prophylactically and therapeutically is described. The method employs humanized antibodies which sequester soluble A? peptide from human biological fluids or which preferably specifically bind an epitope contained within position 13-28 of the amyloid beta peptide A?.