A5086691753- Virus-based gene therapy research
- RNA Interference and Gene Delivery
- RNA Research and Splicing
- Immunotoxicology and immune responses
- Herpesvirus Infections and Treatments
- Neuroblastoma Research and Treatments
- RNA regulation and disease
- Glioma Diagnosis and Treatment
- CRISPR and Genetic Engineering
- DNA Repair Mechanisms
- Veterinary Practice and Education Studies
- Spinal Dysraphism and Malformations
- Cancer Treatment and Pharmacology
- Neurogenetic and Muscular Disorders Research
- Animal Genetics and Reproduction
- Animal testing and alternatives
- Pluripotent Stem Cells Research
Novartis (United States)
2022-2025
Intravenous onasemnogene abeparvovec is approved for spinal muscular atrophy in children <2 years. For later-onset patients, intrathecal may be advantageous over intravenous administration. Recently, microscopic dorsal root ganglion (DRG) changes were observed nonhuman primates (NHPs) following To characterize these DRG findings, two NHP studies evaluating administration conducted: a 12-month study with 6-week interim cohort and 13-week 2-week cohort. The latter investigated the potential...
Hepatotoxicity associated with intravenous/intrathecal adeno-associated virus (AAV) gene therapy has been observed in preclinical species and patients. In nonhuman primates, hepatotoxicity following self-complementary AAV9 administration varies from asymptomatic transaminase elevation minimal to mild microscopic changes symptomatic elevations of liver function thromboinflammatory markers consistent marked hepatocellular necrosis deteriorating clinical condition. These transient acute injury...
The safety of a 2’- O-methoxyethyl antisense oligonucleotide (ASO) was investigated in Mauritius cynomolgus monkeys 41-week Good Laboratory Practice (GLP) toxicity study after multiple intrathecal (IT) administrations. Histopathological examination revealed ectopic formation lymphoid follicles the spinal cord (SC) at injection site all doses and presence granular material neurons SC high-dose animals. seen segments SC, but mainly lumbar segment persisted end 26-week recovery period, while...
Branaplam is a splicing modulator previously under development as therapeutic agent for Spinal Muscular Atrophy Type 1 and Huntington's disease. increased the levels of survival motor neuron protein in preclinical studies was well tolerated early clinical studies; however, peripheral neurotoxicity observed safety study juvenile dogs. The aim this to determine whether serum neurofilament light chain (NfL) concentrations dogs could serve monitoring biomarker branaplam-induced neurotoxicity. A...
In nonhuman primates (NHPs), adeno-associated virus serotype 9 (AAV9) vectorized gene therapy can cause asymptomatic microscopic injury to dorsal root ganglia (DRG) and trigeminal (TG) somatosensory neurons, causing neurofilament light chain (NfL) diffuse into cerebrospinal fluid (CSF) blood. Data from 260 cynomolgus macaques administered vehicle or AAV9 vectors (intrathecally intravenously) were analyzed investigate NfL as a soluble biomarker for monitoring DRG/TG findings. The incidence of...
Veterinary pathology credentials serve as a concise means attesting to educational attainments and experiences indicating readiness for professional practice. Given the cost, time, stress associated with obtaining different qualifications, pathologists must consider what enhance their readiness. In this commentary, authors describe how various degrees certifications have facilitated individual organizational success. The minimum proficient veterinary practice are medical degree (DVM or...