CODON-OPTIMIZED HUMAN NPC1 GENES FOR THE TREATMENT OF NIEMANN-PICK TYPE C1 DEFICIENCY AND RELATED CONDITIONS
US Application 18/816,498
Filed on 2024-08-27
Filed on 2024-08-27
SYNTHETIC MMAB GENES AND AAV VECTORS TO TREAT COBALAMIN B DEFICIENCY
US Application 63/499,013
Filed on 2023-04-28
Filed on 2023-04-28
IMPROVED GENE THERAPY CONSTRUCTS FOR THE TREATMENT OF PROPIONIC ACIDEMIA CAUSED BY MUTATIONS IN PROPIONYL-COA CARBOXYLASE ALPHA
US Application 63/294,511
Filed on 2021-12-29
Filed on 2021-12-29
Novel Vectors and Uses Thereof
US Application 62/907,718
Filed on 2019-09-29
Filed on 2019-09-29
System And Method For Estimating A Quantity Of Interest Based On An Image Of A Histological Section
US Application 62/619,447
Filed on 2018-01-19
Filed on 2018-01-19
LAMP1 THERAPY IN THE TREATMENT OF LYSOSOMAL STORAGE DISORDERS
US Application 63/162,723
Filed on 2021-03-18
Filed on 2021-03-18
METHODS FOR TREATMENT OF SUBJECTS WITH PREEXISTING IMMUNITY TO VIRAL TRANSFER VECTORS
US Application 62/981,564
Filed on 2020-02-26
Filed on 2020-02-26