The present invention relates to transcription regulatory elements (TREs) such as promoters, which may be used to express a transgene within a cell such as a mammalian cell. The invention further relates to polynucleotides and vectors comprising such transcription regulatory elements, which may be operably linked to a transgene, as well as methods of gene therapy based on using such vectors.

The present disclosure relates to CRISPR-Cas10 systems and methods for phage genome editing.

The present disclosure relates to CRISPR-Cas10 systems and methods for phage genome editing.

The present invention relates to a Factor VIII (FVIII) polypeptide, a polynucleotide comprising a Factor VIII nucleotide sequence, and a recombinant AAV construct. The invention further relates to an AAV viral particle comprising the recombinant AAV construct of the invention, and a composition comprising the Factor VIII polypeptide, polynucleotide, recombinant AAV construct or AAV viral particle of the invention. The invention also relates to methods of using, and uses of, the Factor VIII polypeptide, polynucleotide, recombinant AAV construct, AAV viral particle and/or composition of the invention. The invention also relates to uses of the recombinant AAV construct of the invention for the production of AAV viral particles, and methods for producing AAV viral particles using the recombinant AAV constructs of the invention.

The present invention provides Factor VIII polypeptides which comprise one or more substitution mutations compared to a corresponding wild-type Factor VIII, wherein the one or more substitution mutations are located at an inter-domain interface between two domains of the Factor VIII polypeptide. Also provided are polynucleotides comprising a Factor VIII nucleotide sequence encoding a Factor VIII polypeptide of the invention, recombinant AAV constructs comprising such polynucleotides, AAV viral particles comprising such recombinant AAV constructs, compositions comprising the Factor VIII polypeptide, polynucleotide, recombinant AAV construct, or AAV viral particle of the invention, and the use of the Factor VIII polypeptides, polynucleotides, recombinant AAV constructs, AAV viral particles and compositions of the invention in therapy.

A gyroplane employing torque compensated main rotor and hybrid power train is disclosed. The invention incorporates a torque-compensated main rotor system with a common Collective pitch control, which can be driven transiently during flight to allow Vertical Take-Off, Landing and Hovering (VTOLH) flight operations. Torque compensation is via a coaxial counter-rotating (CACR) rotor system, or alternatively using a single rotor in conjunction with one or more electronically-controlled, fixed-pitch, thruster motors. The use of electric motors for lift and torque compensation facilitates electronic and potentially autonomous control of all phases of vertical flight.

A compact, compound gyroplane employing torque compensated main rotor and hybrid power train is disclosed. The invention incorporates a torque-compensated main rotor system with a common Collective pitch control but no Cyclic function, which can be driven transiently during flight to allow vertical take-off, landing and hovering flight operations; torque compensation is via a coaxial counter-rotating (CACR) rotor system, or alternatively using one or more electronically-controlled, fixed-pitch, thruster motors. A mechanical or electro-mechanical hybrid power system allows a single engine to power vertical lift and forward propulsion; the use of electric motors for lift and torque compensation facilitates electronic (and potentially autonomous) control of critical phases of flight.

The disclosure provides for hyaluronic acid functionalized chimeric viral/nonviral nanoparticles, and uses thereof.

The present disclosure relates to CRISPR-Cas10 systems and methods for phage genome editing.

The present disclosure relates to CRISPR-Cas10 systems and methods for phage genome editing.