AAV vectors: The Rubik's cube of human gene therapy

A Pupo, A Fernández, SH Low, A François… - Molecular Therapy, 2022 - cell.com
Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

Current progress and limitations of AAV mediated delivery of protein therapeutic genes and the importance of developing quantitative pharmacokinetic …

EA Chowdhury, G Meno-Tetang, HY Chang… - Advanced Drug Delivery …, 2021 - Elsevier
While protein therapeutics are one of the most successful class of drug molecules, they are
expensive and not suited for treating chronic disorders that require long-term dosing. Adeno …

[HTML][HTML] Systemic administration of novel engineered AAV capsids facilitates enhanced transgene expression in the macaque CNS

AC Stanton, KA Lagerborg, L Tellez, A Krunnfusz… - Med, 2023 - cell.com
Background Adeno-associated virus (AAV) vectors are a promising vehicle for noninvasive
gene delivery to the central nervous system via intravenous infusion. However, naturally …

Gene therapy for neurodegenerative diseases: slowing down the ticking clock

R Martier, P Konstantinova - Frontiers in Neuroscience, 2020 - frontiersin.org
Gene therapy is an emerging and powerful therapeutic tool to deliver functional genetic
material to cells in order to correct a defective gene. During the past decades, several …

Lafora disease: Current biology and therapeutic approaches

S Mitra, E Gumusgoz, BA Minassian - Revue neurologique, 2022 - Elsevier
The ubiquitin system impacts most cellular processes and is altered in numerous
neurodegenerative diseases. However, little is known about its role in neurodegenerative …

[HTML][HTML] Molecular camouflage by a context-specific hydrogel as the key to unlock the potential of viral vector gene therapy

SS Dehnavi, A Cembran, N Mahmoudi… - Chemical Engineering …, 2023 - Elsevier
Gene therapy offers hope for currently untreatable diseases; the patient's own cellular
machinery is recruited to create therapeutics. However, unpredictable responses that lead to …

A CAG repeat-targeting artificial miRNA lowers the mutant huntingtin level in the YAC128 model of Huntington's disease

A Kotowska-Zimmer, L Przybyl, M Pewinska… - … Therapy-Nucleic Acids, 2022 - cell.com
Among the many proposed therapeutic strategies for Huntington's disease (HD), allele-
selective therapies are the most desirable but also the most challenging. RNA interference …

[HTML][HTML] Towards precision therapies for inherited disorders of neurodegeneration with brain iron accumulation

RVV Spaull, AKS Soo, P Hogarth… - Tremor and Other …, 2021 - ncbi.nlm.nih.gov
Background: Neurodegeneration with brain iron accumulation (NBIA) disorders comprise a
group of rare but devastating inherited neurological diseases with unifying features of …

Transcriptome profiling of the ventral pallidum reveals a role for pallido-thalamic neurons in cocaine reward

M Engeln, ME Fox, R Chandra, EY Choi, H Nam… - Molecular …, 2022 - nature.com
Psychostimulant exposure alters the activity of ventral pallidum (VP) projection neurons.
However, the molecular underpinnings of these circuit dysfunctions are unclear. We used …

Kinetics and durability of transgene expression after intrastriatal injection of AAV9 vectors

BS Hollidge, HB Carroll, R Qian, ML Fuller… - Frontiers in …, 2022 - frontiersin.org
Understanding the kinetics and durability of AAV-mediated transgene expression in the
brain is essential for conducting basic neuroscience studies as well as for developing gene …