Disclosed are methods for treating neurological disorders such as Parkinson's disease (PD) using glutamic acid decarboxylase (GAD) and identifying PD patients that will be most receptive to the method of treating PD. In one aspect, the disclosure provides a method of treating PD in a subject in need thereof, the method comprising: (a) identifying a subject having less than about 10 hours, and preferably less than about 8 hours, of on-time per day; and (b) administering to the subject a composition comprising a therapeutically effective amount of one or more vectors to the subthalamic nucleus of the patient, wherein each vector comprises a nucleic acid sequence encoding glutamic acid decarboxylase (GAD) and wherein the subject's on-time is increased.

The present disclosure provides methods of treating a human having a disease or disorder that would benefit from increasing platelet counts. The method involves inhibiting the enzyme activity of biliverdin IXβ reductase (BLVRB) activity or inhibiting the expression of BLVRB gene.

The present invention provides a pharmaceutical composition for treating diabetic retinopathy. According to the present invention, the pharmaceutical composition can treat diabetic retinopathy by means of a single administration and thus, compared to conventional treatment methods, which require intraocular injection on a monthly basis, can reduce pain and ocular damage and infections in patients and reduce treatment costs.

The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia A. In some embodiments, the present disclosure provides methods for dosing a hemophilia A patient with a polynucleotide, e.g., a codon-altered polynucleotide, encoding a Factor VIII polypeptide.

A method to prevent, inhibit or treat one or more symptoms associated with disease of the central nervous system by intranasally, intrathecally, intracerebrovascularly or intravenously administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease, in an amount effective, e.g., to provide for cross-correction.

The present invention relates, in general, to Pompe disease and, in particular, to methods of treating Pompe disease and to compounds/constructs suitable for use in such methods.

The present invention relates, in general, to Pompe disease and, in particular, to a methods of treating Pompe disease and to compounds/constructs suitable for use in such methods.

Provided herein are compositions, systems, kits, and methods for treating a subject, and/or a subject's pre-adipocytes and/or adipocytes, with a composition containing a nucleic acid sequence encoding a protein or other biologically active nucleic acid-encoded molecule (BANEM), or a vector containing the nucleic acid sequence, wherein the treating comprises: a) injecting the composition into one or more subcutaneous (SC) regions of the subject such that one or more protein, or other BANEM, is detectable in a blood, serum, or plasma sample from the subject; and/or b) injecting the composition into one or more SC regions of the subject such that in-vivo transfected pre-adipocytes and/or adipocytes (e.g., transfected cells of fat cell origin) are generated; and/or c) performing the following: i) contacting pre-adipocytes and/or adipocytes (e.g., cells of fat cell origin) from the subject ex-vivo with the composition such that ex-vivo transfected pre-adipocytes and/or adipocytes are generated, and ii) injecting the ex-vivo transfected pre-adipocytes and/or adipocytes into one or more SC regions of the subject.

Provided herein are compositions that include one or more adeno-associated virus (AAVs) vectors and methods of inducing differentiation of a hair cell using these vector(s).

The present invention includes compositions and methods comprising viral vector systems for multiplexed activation of endogenous genes as immunotherapy and viral-based immune-gene therapy.