Abstract: Aspects of the invention described herein concern the incorporation of a FOXP3 cDNA (e.g., full-length human codon-optimized cDNA) into a FOXP3 gene or a non-FOXP3 locus so as to provide constitutive or regulated FOXP3 expression in a primary human CD34+ cells or cells derived from edited CD34+ cells. In some embodiments, guide RNA sequences that are directed to FOXP3, AAVS1, or other candidate loci are used for CRISPR/Cas9-mediated gene regulation, and gene delivery cassettes for HDR based gene-modification are provided.

Abstract: Antibodies, and antigen-binding fragments thereof, that specifically bind to Cluster of Differentiation 1a (CD1a) are provided. Embodiments include uses, and associated methods of using the antibodies, and antigen-binding fragments thereof.

Abstract: A projectile deployment system includes a projectile casing having a pair of sockets, each socket sized to carry one of a pair of anchors of an entangling projectile having a tether connecting the pair of anchors. A pair of independently activable pressure sources are each capable of generating a pressure wave capable of expelling one of the anchors from one of the sockets to deploy the entangling projectile from the projectile casing toward a subject. At least one controller is operable to activate one or both of the pressure sources. Each pressure source is independently activatable at differing times so as to deploy the anchors from the projectile casing at differing times.

Abstract: A projectile deployment system includes a projectile casing having a pair of sockets, each socket sized to carry one of a pair of anchors of an entangling projectile having a tether connecting the pair of anchors. Each of the pair of sockets is angled equally relative to a centerline defined between the sockets. One or more pressure sources is capable of generating a pressure wave capable of expelling one or more of the anchors from the sockets to deploy the entangling projectile from the projectile casing toward a subject. A controller is operable to activate one or both of the pressure sources. A sight is oriented along a target line, the target line being offset relative to the centerline defined between the sockets such that when the anchors are deployed from the projectile casing they exhibit differing flight characteristics.

Abstract: It was discovered that, for individuals with certain types of mutations, clinical outcomes or phenotypes can be very accurately predicted. For example, for an individual with autism harboring a de novo LGD mutation, the patient’s IQ, behavioral phenotypes, and motor/movement phenotypes, and the severity of autism can be predicted. For these LGD mutations, due to a mRNA surveillance mechanism called NMD (nonsense-mediated decay), it was discovered that clinical outcomes and phenotypes are strongly correlated with the expression intensity of the exon harboring the mutation. A method/model was developed, which is called PDS (phenotype dosage sensitivity), to predict phenotypes based on this observation, and the model is able to predict phenotypes at a much higher level of accuracy not previously possible. This disclosure is the first to link LGD mutations and clinical phenotypes in this manner.

Abstract: A projectile deployment system includes an entangling projectile having a pair of anchors and a tether connecting the pellets. A projectile casing includes a pair of sockets, each socket sized to carry one of the pair of anchors. At least one pressure source is capable of expelling one or both of the anchors from the projectile casing toward a subject. At least one of the entangling projectile or the projectile casing can be configured such that the pair of anchors travel toward the subject with differing flight characteristics after being expelled from the projectile casing.

Abstract: A projectile deployment system includes an entangling projectile having a pair of anchors and a tether connecting the pellets. A projectile casing includes a pair of sockets, each socket sized to carry one of the pair of anchors. At least one pressure source is capable of expelling one or both of the anchors from the projectile casing toward a subject. At least one of the entangling projectile or the projectile casing can be configured such that the pair of anchors travel toward the subject with differing flight characteristics after being expelled from the projectile casing.

Abstract: A projectile deployment system includes a projectile casing having a pair of sockets, each socket sized to carry one of a pair of anchors of an entangling projectile having a tether connecting the pair of anchors. Each of the pair of sockets is angled equally relative to a centerline defined between the sockets. One or more pressure sources is capable of generating a pressure wave capable of expelling one or more of the anchors from the sockets to deploy the entangling projectile from the projectile casing toward a subject. A controller is operable to activate one or both of the pressure sources. A sight is oriented along a target line, the target line being offset relative to the centerline defined between the sockets such that when the anchors are deployed from the projectile casing they exhibit differing flight characteristics.

Abstract: Apparatus and method deliver a prosthesis into a hollow body organ or blood vessel. The systems and methods make use of a catheter. A carrier on the distal end of the catheter is sized and configured to carry the prosthesis. A release mechanism and an enclosure mechanism on the distal end are operable to retain and enclose the prosthesis on the carrier. The release mechanism and the enclosure mechanism are also operable to selectively expose and release the prosthesis from the carrier for deployment in the hollow body organ or blood vessel.

Abstract: Devices, systems, and methods for implanting prostheses in the body lumens rely on tacking or anchoring the prostheses with separately introduced fasteners. After initial placement, a fastener applier system is introduced within the expanded prostheses to deploy a plurality of fasteners to at least one prosthesis end. The fasteners are usually helical fasteners which are releasably restrained on the fastener driver, and are delivered by rotation of the fastener driver. The fasteners may be applied singly, typically in circumferentially spaced-apart patterns about the interior of at least one end of the prosthesis. A lumen extension or lumens may be coupled to the prosthesis to extend the reach of the prosthesis within the implantation site. Fasteners may also be applied to the lumen extensions.

Abstract: Devices, systems and methods support tissue in a body organ for the purpose of restoring or maintaining native function of the organ. The devices, systems, and methods do not require invasive, open surgical approaches to be implemented, but, instead, lend themselves to catheter-based, intra-vascular and/or percutaneous techniques.

Abstract: Devices, systems, and methods for implanting radially expandable prostheses in the body lumens rely on tacking or anchoring the prostheses with separately introduced fasteners. The prostheses may be self-expanding or balloon expandable, and may include a single lumen or more than one lumen. After initial placement, a fastener applier system is introduced within the expanded prostheses to deploy a plurality of fasteners to at least one prosthesis end. The fasteners are usually helical fasteners which are releasably restrained on the fastener driver, and are delivered by rotation of the fastener driver. The fasteners may be applied singly, typically in circumferentially spaced-apart patterns about the interior of at least one end of the prosthesis. A lumen extension or lumens may be coupled to the prosthesis to extend the reach of the prosthesis within the implantation site. Fasteners may also be applied to the lumen extensions.

Abstract: Systems and methods introduce and deploy a multi-lumen prosthesis into a blood vessel or hollow body organ. The prosthesis has a trunk divided by an internal septum, which defines, within at least a portion of the trunk interior, a multi-lumen flow channel configuration. A lumen extension component can be sized and configured to be fitted within at least one of the interior lumens to define an extended lumen.

Abstract: Devices, systems and methods support tissue in a body organ for the purpose of restoring or maintaining native function of the organ. The devices, systems, and methods do not require invasive, open surgical approaches to be implemented, but, instead, lend themselves to catheter-based, intra-vascular and/or percutaneous techniques.

Abstract: Systems and methods introduce and deploy a multi-lumen prosthesis into a blood vessel or hollow body organ. The prosthesis has a trunk divided by an internal septum, which defines, within at least a portion of the trunk interior, a multi-lumen flow channel configuration. A lumen extension component can be sized and configured to be fitted within at least one of the interior lumens to define an extended lumen.

Abstract: Apparatus and method deliver a prosthesis into a hollow body organ or blood vessel. The systems and methods make use of a catheter. A carrier on the distal end of the catheter is sized and configured to carry the prosthesis. A release mechanism and an enclosure mechanism on the distal end are operable to retain and enclose the prosthesis on the carrier. The release mechanism and the enclosure mechanism are also operable to selectively expose and release the prosthesis from the carrier for deployment in the hollow body organ or blood vessel.

Abstract: Systems and methods introduce and deploy a multi-lumen prosthesis into a blood vessel or hollow body organ. The prosthesis has a trunk divided by an internal septum, which defines, within at least a portion of the trunk interior, a multi-lumen flow channel configuration. A lumen extension component can be sized and configured to be fitted within at least one of the interior lumens to define an extended lumen.

Abstract: An enclosure for a flash memory hard disk includes a body with a longitudinally defined receiving hole for receiving therein the flash memory hard disk and a radially defined side hole in communication with the receiving hole, a first cover pivotally connected to the body to selectively cover the receiving hole and a lock module received in the side hole for selectively locking the flash memory hard disk in the body.

Abstract: A flow-directed catheter comprises a catheter body having a relatively rigid proximal body segment and a relatively flexible, supple distal body segment. The distal body segment may have a reduced diameter relative to the proximal body segment, at least at its distal tip. Radiopaque markers are provided at the transition between the two body segments and at the tip of the catheter. The radiopaque markers are preferably recessed entirely within the catheter body. Optionally, the distal body segment may have surface irregularities which enhance the ability of the distal body segment to follow blood flow. A flow-directed catheter system comprises the catheter in combination with a coaxial sheath to selectively strengthen at least the proximal of the distal body segment. The catheter may be used for a variety of diagnostic and/or therapeutic procedures.

Abstract: A flow-directed catheter comprises a catheter body having a relatively rigid proximal body segment and a relatively flexible, supple distal body segment. The distal body segment may have a reduced diameter relative to the proximal body segment, at least at its distal tip. Radiopaque markers are provided at the transition between the two body segments and at the tip of the catheter. The radiopaque markers are preferably recessed entirely within the catheter body. Optionally, the distal body segment may have surface irregularities which enhance the ability of the distal body segment to follow blood flow. A flow-directed catheter system comprises the catheter in combination with a coaxial sheath to selectively strengthen at least the proximal of the distal body segment. The catheter may be used for a variety of diagnostic and/or therapeutic procedures.