City of Hope

Other

Homology Medicines, Inc.

Laura Jane Smith

Saswati Chatterjee

Kamehameha Wong

Jason Boke Wright

Jeff Lynn Ellsworth

Hillard Rubin

Kamehameha K Wong, Jr

James Anthony Mcswiggen

James A McSwiggen

Albert Barnes Seymour

Serena Nicole Dollive

Jaime Michelle Prout

Thia Baboval St Martin

Marwa BenHajSalah

Provided herein are adeno-associated virus (AAV) compositions for correcting a mutation in a beta globin gene (HBB) gene and methods of using the same to correct an HBB gene mutation in a cell. Also provided are packaging systems for making the adeno-associated virus compositions.

Adeno-associated virus compositions for restoring HBB gene function and methods of use thereof

Adeno-associated virus (AAV) Clade F vectors or AAV vector variants (relative to AAV9) for precise editing of the genome of a cell and methods and kits thereof are provided. Targeted genome editing using the AAV Clade F vectors or AAV vector variants provided herein occurred at frequencies that were shown to be 1,000 to 100,000 fold more efficient than has previously been reported. Also provided are methods of treating a disease or disorder in a subject by editing the genome of a cell of the subject via transducing the cell with an AAV Clade F vector or AAV vector variant as described herein and further transplanting the transduced cell into the subject to treat the disease or disorder of the subject. Also provided herein are methods of treating a disease or disorder in a subject by in vivo genome editing by directly administering the AAV Clade F vector or AAV vector variant as described herein to the subject.

Adeno-associated virus vector variants for high efficiency genome editing and methods thereof

Provided herein are adeno-associated virus (AAV) compositions that can restore F8 gene function in a cell without co-transducing or co-administering an exogenous nuclease or a nucleotide sequence that encodes an exogenous nuclease. Also provided are methods of using the AAV compositions to correct an F8 gene mutation and/or treat a disease or disorder associated with an F8 gene mutation. Packaging systems for making the adeno-associated virus compositions are also provided.

Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof

Novel adeno-associated virus (AAV) vectors in nucleotide and amino acid forms and uses thereof are provided. The isolates show specific tropism for certain target tissues, such as blood stem cells, liver, heart and joint tissue, and may be used to transduce stem cells for introduction of genes of interest into the target tissues. Certain of the vectors are able to cross tightly controlled biological junctions, such as the blood-brain barrier, which open up additional novel uses and target organs for the vectors, providing for additional methods of gene therapy and drug delivery.

Recombinant adeno-associated vectors for targeted treatment

According to some embodiments, a device to be used with a wheelchair having a leg rest support called pin platform is disclosed. The pin platform includes a plate for supporting a detachable leg rest. The device includes a protection cover member having a longitudinal slit adapted to receive the plate of the leg rest support member.

Wheelchair pin platform guard

Growing community of inventors

Westford, MA, United States of America

Laura Jane Smith