Abstract: The present invention provides in certain embodiments a method of treating autosomal dominant non-syndromic hearing loss (ADNSHL) in a patient in need thereof comprising: (a) identifying a mutation in an ADNSHL-causing gene, (b) preparing a ADNSHL therapeutic miRNA, and (c) administering to the patient a pharmaceutical composition comprising the ADNSHL therapeutic miRNA and a pharmaceutically acceptable carrier.

Abstract: The invention provides novel pyrophosphate synthase inhibitors of formula I and formula II as well salts thereof; the invention also provides compositions comprising such inhibitors and methods for their use.

Abstract: The invention relates to gene polymorphisms and genetic profiles associated with an elevated or a reduced risk of a complement cascade dysregulation disease such as AMD. The invention provides methods and reagents for determination of risk, diagnosis and treatment of such diseases. In an embodiment, the present invention provides methods and reagents for determining sequence variants in the genome of an individual which facilitate assessment of risk for developing such diseases.

Abstract: The invention relates to helminthic parasite preparations and their use for treatment or prevention of GVHD in a subject that has undergone a transplant. The invention also related to helminthic parasite preparations and their use for prevention of GVHD in a subject prior to a transplant.

Abstract: A percutaneous catheter system for use within the human body and an ablation catheter for ablating a selected tissue region within the body of a subject. The percutaneous catheter system can include two catheters that are operatively coupled to one another by magnetic coupling through a tissue structure. The ablation catheter can include electrodes positioned within a central portion. The ablation catheter is positioned such that the central portion of a flexible shaft at least partially surrounds the selected tissue region. Each electrode of the ablation catheter can be activated independently to apply ablative energy to the selected tissue region. The ablation catheter can employ high impedance structures to change the current density at specific points. Methods of puncturing through a tissue structure using the percutaneous catheter system are disclosed. Also disclosed are methods for ablating a selected tissue region using the ablation catheter.

Abstract: The invention provides co-crystals and salts of contrast agents as well as methods to use and prepare the co-crystals and salts.

Abstract: This invention relates to transgenic pig models of cystic fibrosis, cells that can be used to make such animals, and methods of making and using these pigs and cells.

Abstract: The present invention is directed to RNA interference (RNAi) molecules targeted against a nucleic acid sequence, and methods of using these RNAi molecules to reduce off-target toxicity.

Abstract: The present invention relates to optimized aptamers and methods of using these aptamers.

Abstract: GB virus C (GBV-C or hepatitis G virus) is a flavivirus that frequently leads to chronic viremia in humans. The invention provides compositions and methods involving GBV-C E2 polypeptides and peptides for use in modulating immune responses, including inhibition inflammation related to pathogenic T-cell activation.

Abstract: The present invention relates to a rapid detection of microbial-associated nuclease activity with chemically modified nuclease (e.g., ribonuclease) substrates, and probes and compositions useful in detection assays. Accordingly, in certain embodiments, the present invention provides a probe for detecting a microbial endonuclease comprising a substrate oligonucleotide of 2-30 nucleotides in length, a fluorescence-reporter group operably linked to the oligonucleotide, and a fluorescence-quencher group operably linked to the oligonucleotide. The fluorescence-reporter group and the fluorescence-quencher group are separated by at least one RNAse-cleavable residue, e.g., RNA base.

Abstract: Methods and systems for augmentative and alternative communication are disclosed. An example method can comprise receiving a candidate input, classifying the candidate input as an intentional input, and generating a signal in response to the intentional input.

Abstract: The present invention provides three-component compositions comprising microparticles, a tumor antigen, a first immune adjuvant, and a second immune adjuvant. Also provided are chemo-immunotherapeutic compositions comprising microparticles, a chemotherapeutic agent, and an immune adjuvant.

Abstract: This invention provides a device for implantation directly into the spinal cord for the purpose of treating back pain. Electrodes on a backing that conforms directly to the spinal cord are installed as a source of electrical stimulation and pain relief. The electrode array is maintained on the spinal cord by way of a spring or support structure that is anchored to an anatomical structure outside the spinal cord but near the site of implantation. Suitable anchoring structures include the vertebrae and the dura. Secured in this fashion, the support structure maintains a gentle pressure of the electrode array against the spinal cord so as to stay in electrical contact but minimize injury or inflammation. The device may accommodate and buffer movement of the spinal cord both laterally and in a caudal-rostral fashion so that the electrode array remains in place.

Abstract: The present invention is directed to RNA interference (RNAi) molecules targeted against a nucleic acid sequence, and methods of using these RNAi molecules to reduce off-target toxicity.

Abstract: Systems and methods for assessing glaucoma loss using optical coherence topography. One method according to an aspect comprises receiving optical coherence image data and assessing functional glaucoma damage from retinal optical coherence image data. In an aspect, the systems and methods can map regions and layers of the eye to determine structural characteristics to compare to functional characteristics.

Abstract: An adeno-associated virus filler component comprising a nucleic acid of between 3300 and 4200 nucleotides in length is disclosed.

Abstract: The present invention provides a gallium-containing composition for coating/impregnating a device or device surface to prevent biofilm growth formation. The present invention also provides a method of preventing or inhibiting biofilm growth formation. The present invention also provides methods for killing established biofilms.

Abstract: The present invention is directed to RNA interference (RNAi) molecules targeted against a Huntington's disease nucleic acid sequence, and methods of using these RNAi molecules to treat Huntington's disease.

Abstract: A personally portable nanoparticle respiratory deposition (NRD) sampler configured to collect nanoparticles based upon a sampling criterion. In an aspect, the NRD sampler has an impactor stage, and a diffusion stage. In another aspect, the NRD sampler includes a particle size separator in addition to an impactor stage and a diffusion stage.