Crossing the blood-brain barrier with AAV vectors

Systemic AAV vectors for widespread and targeted gene delivery in rodents

(2019) Rosemary C. Challis et al.   Nature Protocols

Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease

(2019) Jeong-A Lim et al.   Molecular Therapy-Methods & Clinical Development

MicroRNAs and Regeneration in Animal Models of CNS Disorders

(2019) Tamara Roitbak   NEUROCHEMICAL RESEARCH

Adeno-associated virus vector as a platform for gene therapy delivery

(2019) Dan Wang et al.   NATURE REVIEWS DRUG DISCOVERY

The GPI-Linked Protein LY6A Drives AAV-PHP.B Transport across the Blood-Brain Barrier

(2019) Juliette Hordeaux et al.   MOLECULAR THERAPY

Astrocyte-selective AAV gene therapy through the endogenous GFAP promoter results in robust transduction in the rat spinal cord following injury

(2019) Jarred M. Griffin et al.   GENE THERAPY

Non‐viral nucleic acid delivery to the central nervous system and brain tumors

(2019) Shanshan Wang et al.   JOURNAL OF GENE MEDICINE

Challenges of gene delivery to the central nervous system and the growing use of biomaterial vectors

(2019) Devan L. Puhl et al.   BRAIN RESEARCH BULLETIN

Gene Therapy for ALS—A Perspective

(2019) Marisa Cappella et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Ly6a Differential Expression in Blood–Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B

(2019) Ana Rita Batista et al.   HUMAN GENE THERAPY

AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains

(2019) Michaël Hocquemiller et al.   Molecular Therapy-Methods & Clinical Development

Viral Strategies for Targeting the Central and Peripheral Nervous Systems

(2018) Claire N. Bedbrook et al.   Annual Review of Neuroscience

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

(2018) Xintao Zhang et al.   BIOMATERIALS

Widespread transduction of astrocytes and neurons in the mouse central nervous system after systemic delivery of a self-complementary AAV-PHP.B vector

(2018) Melvin Y. Rincon et al.   GENE THERAPY

More expansive gene transfer to the rat CNS: AAV PHP.EB vector dose–response and comparison to AAV PHP.B

(2018) Robert D. Dayton et al.   GENE THERAPY

Mapping the Structural Determinants Required for AAVrh.10 Transport across the Blood-Brain Barrier

(2018) Blake H. Albright et al.   MOLECULAR THERAPY

A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates

(2018) Dan Wang et al.   Molecular Therapy-Methods & Clinical Development

Gene therapy for neurological disorders: progress and prospects

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

Emerging Technologies for Delivery of Biotherapeutics and Gene Therapy Across the Blood–Brain Barrier

(2018) Danica B. Stanimirovic et al.   BIODRUGS

Neuro-Coagulopathy: Blood Coagulation Factors in Central Nervous System Diseases

(2017) Ciro De Luca et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

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

(2017) Ken Y Chan et al.   NATURE NEUROSCIENCE

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

Gene therapy for the CNS using AAVs: The impact of systemic delivery by AAV9

(2016) Joana Saraiva et al.   JOURNAL OF CONTROLLED RELEASE

Trafficking of adeno-associated virus vectors across a model of the blood-brain barrier; a comparative study of transcytosis and transduction using primary human brain endothelial cells

(2016) Steven F. Merkel et al.   JOURNAL OF NEUROCHEMISTRY

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

(2016) Jean Bennett et al.   LANCET

In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy

(2016) Sourav R Choudhury 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

Crossing the blood–brain-barrier with viral vectors

(2016) Haiyan Fu et al.   Current Opinion in Virology

Adeno-associated virus serotypes for gene therapeutics

(2015) Leszek Lisowski et al.   CURRENT OPINION IN PHARMACOLOGY

Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats

(2015) J Hordeaux et al.   GENE THERAPY

Gene delivery to the spinal cord using MRI-guided focused ultrasound

(2015) D Weber-Adrian et al.   GENE THERAPY

Central Nervous System and its Disease Models on a Chip

(2015) YoonYoung Yi et al.   TRENDS IN BIOTECHNOLOGY

Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice

(2015) Yannick Tanguy et al.   Frontiers in Molecular Neuroscience

In Vitro and in Vivo Studies on the Transport of PEGylated Silica Nanoparticles across the Blood–Brain Barrier

(2014) Dan Liu et al.   ACS Applied Materials & Interfaces

Adeno-Associated Virus Vectors and Neurological Gene Therapy

(2014) David S. Ojala et al.   NEUROSCIENTIST

Biology of adeno-associated viral vectors in the central nervous system

(2014) Giridhar Murlidharan et al.   Frontiers in Molecular Neuroscience

Safety profile of recombinant adeno-associated viral vectors: focus on alipogene tiparvovec (Glybera®)

(2013) Florence Salmon et al.   Expert Review of Clinical Pharmacology

Targeted Delivery of Self-Complementary Adeno-Associated Virus Serotype 9 to the Brain, Using Magnetic Resonance Imaging-Guided Focused Ultrasound

(2012) Emmanuel Thévenot et al.   HUMAN GENE THERAPY

Gene therapy and neurodevelopmental disorders

(2012) Steven James Gray   NEUROPHARMACOLOGY

Adeno-Associated Virus (AAV) Vectors in the CNS

(2011) Thomas J. McCown   CURRENT GENE THERAPY

Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates

(2011) Lluis Samaranch et al.   HUMAN GENE THERAPY

Preclinical Differences of Intravascular AAV9 Delivery to Neurons and Glia: A Comparative Study of Adult Mice and Nonhuman Primates

(2011) Steven J Gray et al.   MOLECULAR THERAPY

Several rAAV Vectors Efficiently Cross the Blood–brain Barrier and Transduce Neurons and Astrocytes in the Neonatal Mouse Central Nervous System

(2011) Hongwei Zhang et al.   MOLECULAR THERAPY

Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice

(2009) D M McCarty et al.   GENE THERAPY

Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy

(2009) Yong Hong Chen et al.   NATURE MEDICINE

Structure and function of the blood–brain barrier

(2009) N. Joan Abbott et al.   NEUROBIOLOGY OF DISEASE

Gene Therapy Using Adeno-Associated Virus Vectors

(2008) S. Daya et al.   CLINICAL MICROBIOLOGY REVIEWS

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY