Rationale and strategies for the development of safe and effective optimized AAV vectors for human gene therapy

A Srivastava - Molecular Therapy-Nucleic Acids, 2023 - cell.com
Recombinant adeno-associated virus (AAV) vectors have been, or are currently in use, in
332 phase I/II/III clinical trials in a number of human diseases, and in some cases …

[HTML][HTML] Application of hydrogels for targeting cancer stem cells in cancer treatment

F Li, Z Li, C Wei, L Xu, Y Liang, J Yan, Y Li, B He… - Biomedicine & …, 2024 - Elsevier
Cancer stem cells (CSCs) are a major hindrance to clinical cancer treatment. Owing to their
high tumorigenic and metastatic potential, CSCs are vital in malignant tumor initiation …

Co-opting templated aggregation to degrade pathogenic tau assemblies and improve motor function

LVC Miller, G Papa, M Vaysburd, S Cheng… - Cell, 2024 - cell.com
Protein aggregation causes a wide range of neurodegenerative diseases. Targeting and
removing aggregates, but not the functional protein, is a considerable therapeutic challenge …

[HTML][HTML] Immune responses to central nervous system directed adeno-associated virus gene therapy: Does direct CNS delivery make a difference?

AL Harkins, PP Ambegaokar, AM Keeler - Neurotherapeutics, 2024 - Elsevier
Adeno-associated virus (AAV) mediated gene therapy is a leading gene delivery platform
with potential to transform the landscape of treatment for neurological disorders. While AAV …

[HTML][HTML] Recent advances in gene therapy for hemophilia: projecting the perspectives

N Chernyi, D Gavrilova, M Saruhanyan… - Biomolecules, 2024 - mdpi.com
One of the well-known X-linked genetic disorders is hemophilia, which could be hemophilia
A as a result of a mutation in the F8 (factor VIII) gene or hemophilia B as a result of a …

Gene therapy for inherited hearing loss: updates and remaining challenges

R Hahn, KB Avraham - Audiology Research, 2023 - mdpi.com
Hearing loss stands as the most prevalent sensory deficit among humans, posing a
significant global health challenge. Projections indicate that by 2050, approximately 10% of …

Recent advances and future challenges in gene therapy for hearing loss

AE Amariutei, JY Jeng… - Royal Society Open …, 2023 - royalsocietypublishing.org
Hearing loss is the most common sensory deficit experienced by humans and represents
one of the largest chronic health conditions worldwide. It is expected that around 10% of the …

Gene Therapy and kidney diseases

N Tavakolidakhrabadi, WY Ding, MA Saleem… - … Therapy Methods & …, 2024 - cell.com
Chronic kidney disease (CKD) poses a significant global health challenge, projected to
become one of the leading causes of death by 2040. Current treatments primarily manage …

Management of select adverse events following delandistrogene moxeparvovec gene therapy for patients with Duchenne muscular dystrophy

CM Zaidman, NL Goedeker, AA Aqul… - Journal of …, 2024 - journals.sagepub.com
BACKGROUND: Duchenne muscular dystrophy (DMD) is a rare, degenerative, recessive X-
linked neuromuscular disease. Mutations in the gene encoding dystrophin lead to the …

Development of new genome editing tools for the treatment of hyperlipidemia

G Preta - Cells, 2023 - mdpi.com
Hyperlipidemia is a medical condition characterized by high levels of lipids in the blood. It is
often associated with an increased risk of cardiovascular diseases such as heart attacks and …