Abstract: This technology relates to polynucleotides comprising optimized FMR1 open reading frame (ORF) sequences, viral vectors comprising the same, and methods of using the same for delivery of the ORF to a cell or a subject and to treat disorders associated with aberrant expression of FMR1, such as Fragile X syndrome (“FXS”).

Abstract: The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to SURF1 loss and/or misfunction. The methods and compositions of the present disclosure comprise rAAV vectors and rAAV viral vectors comprising transgene nucleic acid molecules comprising nucleic acid sequences encoding for a SURF1 polypeptide.

Abstract: Provide herein are polynucleotides comprising a codon-optimized AP4M1 open reading frame (ORF) sequence encoding an AP4M1 polypeptide, vectors (viral or non-viral vectors) comprising the same, pharmaceutical compositions comprising the same, and methods of using the same for delivery of the polynucleotides to a cell or a subject and to treat AP4M1-related genetic disorders such as AP4M1 deficiency and spastic paraplegia type 50.

Abstract: The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to SLC6A1 loss and/or misfunction. The methods and compositions of the present disclosure comprise rAAV vectors and rAAV viral vectors comprising transgene nucleic acid molecules comprising nucleic acid sequences encoding for a GAT1 polypeptide.

Abstract: The present disclosure provides methods and compositions for the treatment of Angelman Syndrome The methods and compositions of the present disclosure comprise isolated nucleic acid molecules, rAAV vectors and rAAV viral vectors comprising polynucleotide sequences encoding for short hairpin RNA (shRNA) molecules directed against UBE3A-ATS.

Abstract: The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to SLC6A1 loss and/or misfunction. The methods and compositions of the present disclosure comprise rAAV vectors and rAAV viral vectors comprising transgene nucleic acid molecules comprising nucleic acid sequences encoding for a GAT1 polypeptide.

Abstract: The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to MeCP2 loss and/or misfunction, including RETT syndrome. The methods and compositions of the present disclosure comprise rAAV vectors and rAAV viral vectors comprising transgene nucleic acid molecules comprising nucleic acid sequences encoding for a MeCP2 polypeptide.

Abstract: The present disclosure provides methods and compositions for the treatment of diseases and/or disorders in a subject, including, but not limited to neurological disorders such as giant axonal neuropathy. The methods described herein include direct administration of a gene therapy (e.g. an rAAV viral vector) to a subject via injection into a vagus nerve (e.g. the left vagus nerve) of the subject.

Abstract: The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to MeCP2 loss and/or misfunction, including RETT syndrome. The methods and compositions of the present disclosure comprise rAAV vectors and rAAV viral vectors comprising transgene nucleic acid molecules comprising nucleic acid sequences encoding for a MeCP2 polypeptide.

Abstract: The present disclosure provides methods and compositions for the treatment of diseases and/or disorders in a subject, including, but not limited to neurological disorders such as giant axonal neuropathy. The methods described herein include direct administration of a gene therapy (e.g. an rAAV viral vector) to a subject via injection into a vagus nerve (e.g. the left vagus nerve) of the subject.

Abstract: A system and method for estimating vascular flow using CT imaging include a computer readable storage medium having stored thereon a computer program comprising instructions, which, when executed by a computer, cause the computer to acquire a first set of data comprising anatomical information of an imaging subject, the anatomical information comprises information of at least one vessel. The instructions further cause the computer to process the anatomical information to generate an image volume comprising the at least one vessel, generate hemodynamic information based on the image volume, and acquire a second set of data of the imaging subject. The computer is also caused to generate an image comprising the hemodynamic information in combination with a visualization based on the second set of data.

Abstract: Methods for treating IBD or an IBD related disorder in a subject in need thereof are provided that comprise combined intrathecal administration of a polynucleotide comprising a CLN1 open reading frame and intravenous administration of the polynucleotide. The polynucleotide comprising the CLN1 open reading frame is a wild-type CLN1 polynucleotide. In another aspect, the polynucleotide comprising the CLN1 open reading frame comprises codon-optimized polynucleotide sequence of the polynucleotide or its complement and is codon-optimized for expression in a human cell.

Abstract: The present disclosure provides methods and compositions for the treatment of diseases and/or disorders in a subject, including, but not limited to neurological disorders such as giant axonal neuropathy. The methods described herein include direct administration of a gene therapy (e.g. an rAAV viral vector) to a subject via injection into a vagus nerve (e.g. the left vagus nerve) of the subject.

Abstract: The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to MeCP2 loss and/or misfunction, including RETT syndrome. The methods and compositions of the present disclosure comprise rAAV vectors and rAAV viral vectors comprising transgene nucleic acid molecules comprising nucleic acid sequences encoding for a MeCP2 polypeptide.

Abstract: The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to SURF1 loss and/or misfunction. The methods and compositions of the present disclosure comprise rAAV vectors and rAAV viral vectors comprising transgene nucleic acid molecules comprising nucleic acid sequences encoding for a SURF1 polypeptide.

Abstract: The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to SLC6A1 loss and/or misfunction. The methods and compositions of the present disclosure comprise rAAV vectors and rAAV viral vectors comprising transgene nucleic acid molecules comprising nucleic acid sequences encoding for a GAT1 polypeptide.

Abstract: A system and method for estimating vascular flow using CT imaging include a computer readable storage medium having stored thereon a computer program comprising instructions, which, when executed by a computer, cause the computer to acquire a first set of data comprising anatomical information of an imaging subject, the anatomical information comprises information of at least one vessel. The instructions further cause the computer to process the anatomical information to generate an image volume comprising the at least one vessel, generate hemodynamic information based on the image volume, and acquire a second set of data of the imaging subject. The computer is also caused to generate an image comprising the hemodynamic information in combination with a visualization based on the second set of data.

Abstract: A system and method for estimating vascular flow using CT imaging include a computer readable storage medium having stored thereon a computer program comprising instructions, which, when executed by a computer, cause the computer to acquire a first set of data comprising anatomical information of an imaging subject, the anatomical information comprises information of at least one vessel. The instructions further cause the computer to process the anatomical information to generate an image volume comprising the at least one vessel, generate hemodynamic information based on the image volume, and acquire a second set of data of the imaging subject. The computer is also caused to generate an image comprising the hemodynamic information in combination with a visualization based on the second set of data.

Abstract: A system and method for estimating vascular flow using CT imaging include a computer readable storage medium having stored thereon a computer program comprising instructions, which, when executed by a computer, cause the computer to acquire a first set of data comprising anatomical information of an imaging subject, the anatomical information comprises information of at least one vessel. The instructions further cause the computer to process the anatomical information to generate an image volume comprising the at least one vessel, generate hemodynamic information based on the image volume, and acquire a second set of data of the imaging subject. The computer is also caused to generate an image comprising the hemodynamic information in combination with a visualization based on the second set of data.

Abstract: The present invention is a detection system and method for using the detection system in radiant energy imaging systems. In particular, the present invention is an improved detection system employing multiple screens for greater detection efficiency. And more particularly, the present invention is a detection system for detecting electromagnetic radiation having an enclosure having four adjacent walls, connected to each other at an angle and forming a rectangle and interior portion of the enclosure, a front side area and a back side area formed from the four adjacent walls and located at each end of the enclosure, at least two screens, that further include an active area for receiving and converting electromagnetic radiation into light, and a photodetector, positioned in the interior portion of the enclosure, having an active area responsive to the light.