Targeted Integration Into The Albumin (Alb) Locus As A Treatment For Hereditary Methylmalonic Acidemia (MMA) Caused By Methylmalonyl-coA Mutase (MUT) Deficiency
US Application 62/177,771
Filed on 2018-08-10
Filed on 2018-08-10
DETECTING PROTEIN INTERACTION SITES IN NUCLEIC ACIDS
US Application 62/615,233
Filed on 2018-01-09
Filed on 2018-01-09
ISOTOPIC BIOMARKERS OF ORGANIC ACIDEMIAS
US Application 62/579,247
Filed on 2017-10-31
Filed on 2017-10-31
Biomarkers of Organic Acidemias
US Application 62/556,071
Filed on 2017-09-08
Filed on 2017-09-08
Novel Codon-optimized And Tagged Human NPC1 Genes And AAV Vectors For The Treatment Of Niemann-Pick Type C1 Deficiency And Related Conditions
US Application 62/522,677
Filed on 2017-06-20
Filed on 2017-06-20
METHODS OF DETECTING AND TREATING GATM-RELATED RENAL DISORDERS
US Application 62/515,834
Filed on 2017-06-06
Filed on 2017-06-06
CANCER DETECTION AND CLASSIFICATION
US Application 62/898,670
Filed on 2019-09-11
Filed on 2019-09-11
OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA
US Application 62/330,027
Filed on 2016-04-29
Filed on 2016-04-29
Gene Therapy For Combined Methylmalonic Acidemia/Aciduria and Hyperhomocysteinemia/Homocystinuria, Cobalamin C Type, and Deficiency of MMACHC
US Application 62/279,285
Filed on 2016-01-15
Filed on 2016-01-15
Gene Therapy For Cobalamin C Deficiency
US Application 62/266,352
Filed on 2015-12-11
Filed on 2015-12-11