Customized blood-brain barrier shuttle peptide to increase AAV9 vector crossing the BBB and augment transduction in the brain

DPepH3, an Improved Peptide Shuttle for Receptor-independent Transport across the Blood-Brain Barrier

(2020) Marco Calvinho Cavaco et al.   CURRENT PHARMACEUTICAL DESIGN

Novel cyclic peptides facilitating transcellular blood-brain barrier transport of macromolecules in vitro and in vivo

(2020) Shunsuke Yamaguchi et al.   JOURNAL OF CONTROLLED RELEASE

Intrastromal Gene Therapy Prevents and Reverses Advanced Corneal Clouding in a Canine Model of Mucopolysaccharidosis I

(2020) Keiko Miyadera et al.   MOLECULAR THERAPY

Advances in therapeutic peptides targeting G protein-coupled receptors

(2020) Anthony P. Davenport et al.   NATURE REVIEWS DRUG DISCOVERY

Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10

(2020) Lalitha R. Belur et al.   Molecular Genetics and Metabolism Reports

Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors

(2019) Hildegard Büning et al.   Molecular Therapy-Methods & Clinical Development

Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

(2019) Allison M. Keeler et al.   Annual Review of Virology

Blood–Brain Barrier- and Blood–Brain Tumor Barrier-Penetrating Peptide-Derived Targeted Therapeutics for Glioma and Malignant Tumor Brain Metastases

(2019) Long Chen et al.   ACS Applied Materials & Interfaces

The Influence of Murine Genetic Background in Adeno-Associated Virus Transduction of the Mouse Brain

(2019) Ting He et al.   Human Gene Therapy Clinical Development

Blood-brain barrier shuttle peptides enhance AAV transduction in the brain after systemic administration

(2018) Xintao Zhang et al.   BIOMATERIALS

Therapeutic peptides: Historical perspectives, current development trends, and future directions

(2018) Jolene L. Lau et al.   BIOORGANIC & MEDICINAL CHEMISTRY

The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice

(2018) Juliette Hordeaux et al.   MOLECULAR THERAPY

Gene therapy for neurological disorders: progress and prospects

(2018) Benjamin E. Deverman et al.   NATURE REVIEWS DRUG DISCOVERY

Phage-displayed peptides targeting specific tissues and organs

(2018) Josu Andrieu et al.   JOURNAL OF DRUG TARGETING

Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I

(2017) Lalitha R. Belur et al.   HUMAN GENE THERAPY

Characterization of Adeno-Associated Viral Vector-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice

(2017) Jenny A. Greig et al.   HUMAN GENE THERAPY

Application of polyploid adeno-associated virus vectors for transduction enhancement and neutralizing antibody evasion

(2017) Zheng Chai et al.   JOURNAL OF CONTROLLED RELEASE

Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems

(2017) Ken Y Chan et al.   NATURE NEUROSCIENCE

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

(2017) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Development of next generation adeno-associated viral vectors capable of selective tropism and efficient gene delivery

(2016) Chuanling Zhang et al.   BIOMATERIALS

Blood–brain barrier shuttle peptides: an emerging paradigm for brain delivery

(2016) Benjamí Oller-Salvia et al.   CHEMICAL SOCIETY REVIEWS

A brain microvasculature endothelial cell‐specific viral vector with the potential to treat neurovascular and neurological diseases

(2016) Jakob Körbelin et al.   EMBO Molecular Medicine

The potential use of rabies virus glycoprotein-derived peptides to facilitate drug delivery into the central nervous system: a mini review

(2016) Rachel Huey et al.   JOURNAL OF DRUG TARGETING

Pulmonary Targeting of Adeno-associated Viral Vectors by Next-generation Sequencing-guided Screening of Random Capsid Displayed Peptide Libraries

(2016) Jakob Körbelin et al.   MOLECULAR THERAPY

Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

(2016) Benjamin E Deverman et al.   NATURE BIOTECHNOLOGY

AAV Gene Therapy for MPS1-associated Corneal Blindness

(2016) Melisa Vance et al.   Scientific Reports

dNP2 is a blood–brain barrier-permeable peptide enabling ctCTLA-4 protein delivery to ameliorate experimental autoimmune encephalomyelitis

(2015) Sangho Lim et al.   Nature Communications

Synergistic Dual-Ligand Doxorubicin Liposomes Improve Targeting and Therapeutic Efficacy of Brain Glioma in Animals

(2014) Taili Zong et al.   MOLECULAR PHARMACEUTICS

Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates

(2014) Marinee K Chuah et al.   MOLECULAR THERAPY

Systemic gene delivery to the central nervous system using Adeno-associated virus

(2014) Mathieu Bourdenx et al.   Frontiers in Molecular Neuroscience

LDLR-mediated peptide-22-conjugated nanoparticles for dual-targeting therapy of brain glioma

(2013) Bo Zhang et al.   BIOMATERIALS

Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS

(2013) Kevin D Foust et al.   MOLECULAR THERAPY

The advent of AAV9 expands applications for brain and spinal cord gene delivery

(2012) Robert D Dayton et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

TerminalN-Linked Galactose Is the Primary Receptor for Adeno-associated Virus 9

(2011) Shen Shen et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

A Potential Role of Distinctively Delayed Blood Clearance of Recombinant Adeno-associated Virus Serotype 9 in Robust Cardiac Transduction

(2011) Nicole M Kotchey et al.   MOLECULAR THERAPY

Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

(2010) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

Systemic Delivery of scAAV9 Expressing SMN Prolongs Survival in a Model of Spinal Muscular Atrophy

(2010) C. F. Valori et al.   Science Translational Medicine

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY