Abstract: The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD). In particular embodiments, the invention provides methods for correcting mutations in a beta globin polynucleotide using modified adenosine base editors termed “ABE8” having unprecedented levels (e.g., >60-70%) of efficiency.

Abstract: The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD). In particular embodiments, the invention provides methods for correcting mutations in a beta globin polynucleotide using modified adenosine base editors termed “ABE8” having unprecedented levels (e.g., >60-70%) of efficiency.

Abstract: The disclosure provides compositions comprising novel adenosine base editors (e.g., ABE8) that have increased efficiency and methods of using these adenosine deaminase variants for editing a target sequence.

Abstract: The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD). In particular embodiments, the invention provides methods for correcting mutations in a beta globin polynucleotide using modified adenosine base editors termed “ABE8” having unprecedented levels (e.g., >60-70%) of efficiency.

Abstract: The invention provides compositions comprising novel adenosine base editors (e.g., ABE8) that have increased efficiency and methods of using base editors comprising adenosine deaminase variants for altering mutations associated with Glycogen Storage Disease Type 1a (GSD1a).

Abstract: The invention features compositions and methods for treating, reducing, or ameliorating the debilitating effects of Amyotrophic Lateral Sclerosis (ALS) and spinal and bulbar muscular atrophy (SBMA). Provided herein are compositions and methods of using improved new base editors (e.g., adenosine base editors) comprising a polynucleotide programmable nucleotide binding domain and a nucleobase editing domain in conjunction with a guide polynucleotide to disrupt normal transcription of a gene associated with a genetic disease or condition, e.g. ALS, or SBMA by modifying a target gene associated with the genetic disorder or condition with a base editor system provided herein.

Abstract: The invention features compositions and methods for altering mutations associated with Rett Syndrome (RTT). Provided herein are compositions and methods of using base editors comprising a polynucleotide programmable nucleotide binding domain and a nucleobase editing domain in conjunction with a guide polynucleotide. Also provided herein are base editor systems for editing nucleobases of target nucleotide sequences.

Abstract: Provided herein are compositions and methods of using base editors comprising a polynucleotide programmable nucleotide binding domain and a nucleobase editing domain in conjunction with a guide polynucleotide. Also provided herein are base editor systems for editing nucleobases of target nucleotide sequences.

Abstract: The disclosure provides compositions comprising novel adenosine base editors (e.g., ABE8) that have increased efficiency and methods of using these adenosine deaminase variants for editing a target sequence.

Abstract: The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD). In particular embodiments, the invention provides methods for correcting mutations in a beta globin polynucleotide using modified adenosine base editors termed “ABE8” having unprecedented levels (e.g., >60-70%) of efficiency.

Abstract: The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD). In particular embodiments, the invention provides methods for correcting mutations in a beta globin polynucleotide using modified adenosine base editors termed “ABE8” having unprecedented levels (e.g., >60-70%) of efficiency.

Abstract: The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD). In particular embodiments, the invention provides methods for correcting mutations in a beta globin polynucleotide using modified adenosine base editors termed “ABE8” having unprecedented levels (e.g., >60-70%) of efficiency.

Abstract: The disclosure provides compositions comprising novel adenosine base editors (e.g., ABE8) that have increased efficiency and methods of using these adenosine deaminase variants for editing a target sequence.

Abstract: A pedal for a percussion instrument, such as a bass drum, has a base plate and a foot plate pivotally mounted the base plate which is operably connected to an instrument striker. The pedal either has no heel plate or has a very short heel guide and is adapted so that when the tip of the player's foot is at an optimal position or “sweet spot” on the foot plate, the player's heel extends to the playing surface.

Abstract: A pedal for a percussion instrument, such as a bass drum, has a base plate and a foot plate pivotally mounted the base plate which is operably connected to an instrument striker. The pedal either has no heel plate or has a very short heel guide and is adapted so that when the tip of the player's foot is at an optimal position or “sweet spot” on the foot plate, the player's heel extends to the playing surface.

Abstract: Luminaire optical assemblies intended for pole-mounted and similar applications and capable of effective I.E.S. cutoff performance while exhibiting desirable light distributions even with vertically oriented lamping, the invention contemplates in primary embodiments light-transmissive reflector/refractor combinations typically formed of glass, acrylics and the like, uplight shielding and reflector assemblies of a variety of configurations being disposed within the reflector/refractor combinations to produce desired cutoff characteristics while preserving daytime appearance during nocturnal operation. In certain embodiments of the invention, uplight is controlled by the several variations of the uplight shielding and reflector assemblies to cause light-transmissive portions of the reflector/refractor combinations to subtly glow and thus produce a desired appearance while also producing cutoff characteristics necessary to reduce urban sky glow and glare.

Abstract: Lighting fixtures for illuminating vertical surfaces of predetermined dimensions such as billboards or similar signs, the invention in several embodiments takes the form of primary reflector structures capable of directing light from a light source disposed within each of said fixtures either directly to the vertical surface or to refractive structures located on the fixtures for redirection of light to the vertical surface for even illumination of said surface with minimal light pollution from “spill” light. The primary reflector structures of the invention are curvilinear in conformation and extend from above a light source to a location between the source and the vertical surface, the reflector structures being smoothly contoured or formed into a plurality of reflective segments.

Abstract: An optical interface for coupling a refractor and a reflector in a luminaire assembly includes a flange having an inner surface and an outer surface, the outer surface having reflecting means and disposed adjacent an air gap, the flange having a shape which works in concert with the reflector and is adapted to be affixable to the reflector and the refractor.