The present invention relates to recombinant adeno-associated virus (rAAV) delivery of an alpha-sarcoglycan gene. The invention provides rAAV products and methods of using the rAAV in the treatment of limb girdle muscular dystrophies such as LGMD2D.

The present invention relates to a system (100) for extracorporeal blood treatment comprising a first inlet (1) for introducing a bloodstream to be treated into the system (100), three blood treatment apparatus (A, D, G), as well as an outlet (2) for discharging a treated bloodstream from the system (100), wherein the system comprises an adsorber apparatus (A) and/or a plasma separator apparatus, a dialysis apparatus (D) and a gas exchange apparatus (G), and wherein the three blood treatment apparatus (A, D, G) are sequentially connected in series in a functional state of system (100) application between the inlet (1) and the outlet (2) of the system relative to a direction of blood flow of a bloodstream to be treated and can be consecutively perfused extracorporeally by a bloodstream to be treated. The present invention further relates to a treatment apparatus comprising such a system, a kit comprising the components of such a system, a method for operating such a system (100) as well as a method for extracorporeal blood treatment with such a system (100).

The invention provides a method, apparatus and system for separating blood and other types of cellular components, and can be combined with holographic optical trapping manipulation or other forms of optical tweezing. One of the exemplary methods includes providing a first flow having a plurality of blood components; providing a second flow; contacting the first flow with the second flow to provide a first separation region; and differentially sedimenting a first blood cellular component of the plurality of blood components into the second flow while concurrently maintaining a second blood cellular component of the plurality of blood components in the first flow. The second flow having the first blood cellular component is then differentially removed from the first flow having the second blood cellular component. Holographic optical traps may also be utilized in conjunction with the various flows to move selected components from one flow to another, as part of or in addition to a separation stage.

Disclosed are methods and systems for a body-fluid (e.g., blood) treatment. The methods and systems include (a) conveying a volume of body-fluid (e.g., blood) via a first conduit from a vascular access of a patient to a blood chamber at a first flow rate, the first conduit having only a single lumen; (b) conveying the body-fluid (e.g., blood) from the blood chamber through a filtration device at a second flow rate to perform an extracorporeal treatment on the blood and returning the treated blood to the blood chamber; and (c) returning the body-fluid (e.g., blood) from the blood chamber to the vascular access of the patient at a third flow rate via the first conduit, wherein the second flow rate is decoupled from both the first and third flow rates.

Embodiments of the present invention provide an extra corporeal membrane oxygenation circuit, wherein a pump communicates blood from a patient to an oxygenator and thence back to the patient, comprising: (a) a medium diameter venous line configured to accept blood from the patient and communicate the blood to the pump; (b) a medium diameter arterial line configured to accept blood from the oxygenator and communicate the blood to the patient; (c) one or more shunts connected in a series, where each shunt comprises a medium diameter input connected to a medium diameter output, where the medium diameter output is configured to connect to a medium diameter input of a successive shunt; a small diameter outlet between the medium diameter input and the medium diameter output; and a stopcock connected to the small diameter output such that flow out of the small diameter outlet can be controlled by the stopcock; wherein a first of such shunts is connected to accept blood from the venous line in parallel with the pump and wherein a last of such shunts is connected to communicate blood to the arterial line.

The invention provides a method, apparatus and system for separating blood and other types of cellular components, and can be combined with holographic optical trapping manipulation or other forms of optical tweezing. One of the exemplary methods includes providing a first flow having a plurality of blood components; providing a second flow; contacting the first flow with the second flow to provide a first separation region; and differentially sedimenting a first blood cellular component of the plurality of blood components into the second flow while concurrently maintaining a second blood cellular component of the plurality of blood components in the first flow. The second flow having the first blood cellular component is then differentially removed from the first flow having the second blood cellular component. Holographic optical traps may also be utilized in conjunction with the various flows to move selected components from one flow to another, as part of or in addition to a separation stage.

The invention provides a method, apparatus and system for separating blood and other types of cellular components, and can be combined with holographic optical trapping manipulation or other forms of optical tweezing. One of the exemplary methods includes providing a first flow having a plurality of blood components; providing a second flow; contacting the first flow with the second flow to provide a first separation region; and differentially sedimenting a first blood cellular component of the plurality of blood components into the second flow while concurrently maintaining a second blood cellular component of the plurality of blood components in the first flow. The second flow having the first blood cellular component is then differentially removed from the first flow having the second blood cellular component. Holographic optical traps may also be utilized in conjunction with the various flows to move selected components from one flow to another, as part of or in addition to a separation stage.

Dialysis systems are disclosed comprising new fluid flow circuits. Systems may include blood and dialysate flow paths, where the dialysate flow path includes balancing, mixing, and/or directing circuits. Dialysate preparation may be decoupled from patient dialysis. Circuits may be defined within one or more cassettes. The fluid circuit fluid flow paths may be isolated from electrical components. A gas supply in fluid communication with the dialysate flow path and/or the dialyzer able to urge dialysate through the dialyzer and urge blood back to the patient may be included for certain emergency situations. Fluid handling devices, such as pumps, valves, and mixers that can be actuated using a control fluid may be included. Control fluid may be delivered by an external pump or other device, which may be detachable and/or generally rigid, optionally with a diaphragm dividing the device into first and second compartments.

A method for filling and venting a device for extracorporeal blood treatment is disclosed, such as a patient module in a heart-lung machine, without attached patient. A filling liquid from a filling liquid container located higher than the device flows by gravity via a venous side of the system into a reservoir and flows onwards into a blood pump located at the lower end of the reservoir, wherein a first controllable valve (HC1) for a venting line of a filter is opened and, after the response of an upper filling level sensor in the reservoir, is closed. An upper level of the filter is positioned higher than the upper filling level sensor, and a start-stop motion of the blood pump is performed, as a result of which a stepped flooding of the filter is made providing for an advantageous de-airing of the device.

The invention relates to a system, and the associated method for using said system, for treating haemorrhagic fluid previously taken from a patient for the purpose of autotransfusion, comprising a unit for treating (100) haemorrhagic fluid, said treatment unit (100) comprising: a filtration device (110) for tangential filtration comprising a filtration membrane (113) arranged in a housing (114) so as to separate an intake chamber (111) from a discharge chamber (112), the intake chamber (111) and the discharge chamber (112) each having an inlet (111a; 112a) and an outlet (111b; 112b) for fluids; a treatment pouch (140) having an inlet (140a) and an outlet (140b) fluidically connected by a recirculation line (150) to the outlet (111b) and to the inlet (111a) of the intake chamber (111) of the filtration device (110), respectively, allowing haemorrhagic fluid to circulate in the recirculation line (150) in a direction going from the outlet (140b) of the treatment pouch (140) to the inlet (140a) of the treatment pouch (140) through the intake chamber (111) of the filtration device (110); a cleaning line (180) fluidically connected to the inlet (112a) of the discharge chamber (112) of the filtration device (110) to convey cleaning fluid into said discharge chamber (112); and a first flow regulation member (181) arranged to regulate the flow in the cleaning line (180) and a second flow regulation member (131) arranged to regulate the flow in a discharge line (130) fluidically connected to the outlet (112b) of the discharge chamber (112) of the filtration device (110), so as to be able to control the pressure of cleaning fluid in the discharge chamber (112).