Discover open access scientific publications

Search, annotate, share and cite publications

Publications by E Kakkis

Intrathecal Administration of AAV Vectors for the Treatment of Lysosomal Storage in the Brains of MPS I Mice

Gene Therapy
Molecular MedicineGeneticsMolecular Biology
2006English

Related publications

681. rDNA-AAV Vectors for the Treatment of Phenylketonuria

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2010English

Whole Genome Sequencing for Mutation Discovery in a Single Case of Lysosomal Storage Disease (MPS Type 1) in the Dog

Scientific Reports
Multidisciplinary
2020English

604. Intrathecal AAV9-Mediated Gene Delivery Corrects Lysosomal Storage Throughout the CNS in a Large Animal Model of Mucopolysaccharidosis Type I

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

The Molecular Basis of Lysosomal Storage Diseases and Their Treatment

Biochemical Society Transactions
Biochemistry
2000English

The Natural History of MPS I: Global Perspectives From the MPS I Registry

Genetics in Medicine
MedicineGenetics
2014English

Assaying the Stability and Inactivation of AAV Serotype 1 Vectors

Human gene therapy methods
Applied MicrobiologyBiotechnologyGeneticsPharmacologyMolecular Medicine
2017English

The Intrathecal Treatment of Tabes

BMJ
1914English

Growth Patterns for Untreated Individuals With MPS I: Report From the International MPS I Registry

American Journal of Medical Genetics, Part A
Genetics
2019English

221. The Utilization of Capsid-Optimized Adeno-Associated Virus (AAV) Vectors for Cancer Immunotherapy

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2015English

Amanote Research

Note-taking for researchers

Follow Amanote

© 2024 Amaplex Software S.P.R.L. All rights reserved.

Privacy PolicyRefund Policy