The present invention relates to an AAV vector carrying a predetermined hybrid HGF gene sequence. Use of the AAV vector of the present invention allows a hybrid HGF gene to be delivered to a subject at a high delivery yield.

Disclosed herein are systems and methods for presenting mixed reality audio. In an example method, audio is presented to a user of a wearable head device. A first position of the user's head at a first time is determined based on one or more sensors of the wearable head device. A second position of the user's head at a second time later than the first time is determined based on the one or more sensors. An audio signal is determined based on a difference between the first position and the second position. The audio signal is presented to the user via a speaker of the wearable head device. Determining the audio signal comprises determining an origin of the audio signal in a virtual environment. Presenting the audio signal to the user comprises presenting the audio signal as if originating from the determined origin. Determining the origin of the audio signal comprises applying an offset to a position of the user's head.

The present disclosure provides novel vectors and methods useful in treating genetic diseases, brain disorders, and neurological diseases and disorders, including gene therapy vectors and methods of administering such to a subject in need thereof.

Disclosed herein are compositions and pharmaceutical formulations that comprise a binding moiety conjugated to a polynucleic acid molecule and a polymer. Also described herein include methods for treating a cancer which utilize a composition or a pharmaceutical formulation comprising a binding moiety conjugated to a polynucleic acid molecule and a polymer.

The present invention provides viral vectors for use in the treatment and prevention of diseases associated with Schwann cells by delivering polynucleotides specifically to Schwann cells and achieving Schwann cell specific expression. The present invention has particular application in treatment and prevention of Charcot-Marie-Tooth disease and other demyelinating neuropathies. The preferred vectors are adeno-associated vectors (AAV) having a Schwann cell-specific promoter from the Myelin Protein Zero (Mpz, P0) or a minimal Mpz promoter.

In some embodiments herein, methods, compositions, and uses for modulating lymphatic vessels of the central nervous system are described. In some embodiments, methods, compositions, or uses for treating, preventing, or ameliorating symptoms of a neurological disease comprise increasing flow via meningeal lymphatic vessels are described.

The present invention provides, among other things, an improved method of treating cystic fibrosis (CF) in a human subject. The method comprises administering a composition comprising an mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein at a concentration of 0.5 mg/mL or greater to a human subject via nebulization. The composition is aerosolized using a nebulizer and a nominal dose of the mRNA is administered to the human subject via the nebulizer over a period of time, typically at least 30 minutes, at a suitable nebulization rate, e.g., at least 0.2 mL/minute.

Compositions including modified adeno-associated virus (AAV) vectors comprising a recombinant AAV (rAAV)-based genome are provided herein, wherein the rAAV-based genome includes one or more of: a brain derived neurotrophic factor (BDNF)-encoding cDNA insert; or a neurotrophin-3 (NT-3)-encoding cDNA insert. Also provided are methods of treating motor neuron degenerative disorders, such as amyotrophic lateral sclerosis (ALS), by administering the disclosed compositions.

Disclosed herein are therapeutic agents capable of increasing the expression level of ELOVL2. Also described herein are therapeutic agents that reduce or slow an aging phenotype. Methods for treating age-related eye diseases or conditions are also provided. Methods for treating an age-related eye disease or condition in a subject by administering one or more therapeutic agents are provided.

Provided is the intravitreal dosing of recombinant adeno-associated virus (rAAV)-based gene therapies for the treatment of color vision deficiencies such as Blue Cone Monochromacy (BCM) and Red-Green Color Blindness.