Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2

2 Dec 2020

Abstract excerpt

< 0.0001), respectively. Intraparenchymal brain administration of AAVrh.10hCLN2 slowed the progression of disease in children with CLN2 disease. However, improvements in vector design and delivery strategies will be necessary to halt disease progression using gene therapy.

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Cytomegalovirus and herpesvirus research
Share to Lysosomal Storage Disorders Research
Share to RNA regulation and disease

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 33268510
DOI: 10.1126/scitranslmed.abb5413

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2PMID 33268510Read ↗

AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCLSemantic proximity Read ↗

Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector.Semantic proximity Read ↗

Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten DiseaseSemantic proximity Read ↗

Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 diseaseSemantic proximity Read ↗

Long-term safety and dose escalation of intracerebroventricular CLN5 gene therapy in sheep supports clinical translation for CLN5 Batten diseaseSemantic proximity Read ↗

AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten DiseaseSemantic proximity Read ↗

Clinical protocol. Administration of a replication-deficient adeno-associated virus gene transfer vector expressing the human CLN2 cDNA to the brain of children with late infantile neuronal ceroid lipofuscinosis.Semantic proximity Read ↗

Longitudinal In Vivo Monitoring of the CNS Demonstrates the Efficacy of Gene Therapy in a Sheep Model of CLN5 Batten DiseaseSemantic proximity Read ↗

AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten diseaseSemantic proximity Read ↗

AAV8-mediated Hepatic Gene Transfer in Infant Rhesus Monkeys (Macaca mulatta)Semantic proximity Read ↗

Safety of Direct Administration of AAV2 <sub>CU</sub> hCLN2, a Candidate Treatment for the Central Nervous System Manifestations of Late Infantile Neuronal Ceroid Lipofuscinosis, to the Brain of Rats and Nonhuman PrimatesSemantic proximity Read ↗

Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis.Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.