rAAV immunogenicity, toxicity, and durability in 255 clinical trials: A meta-analysis
Recombinant Adeno-associated virus (rAAV) is one of the main delivery vectors for gene
therapy. To assess immunogenicity, toxicity, and features of AAV gene therapy in clinical …
therapy. To assess immunogenicity, toxicity, and features of AAV gene therapy in clinical …
Immunogenicity of recombinant adeno-associated virus (AAV) vectors for gene transfer
Recombinant adeno-associated viruses (AAVs) have emerged as promising gene delivery
vehicles resulting in three US Food and Drug Administration (FDA) and one European …
vehicles resulting in three US Food and Drug Administration (FDA) and one European …
Gene therapy approaches for the treatment of hemophilia B
AB Soroka, SG Feoktistova, ON Mityaeva… - International Journal of …, 2023 - mdpi.com
In contrast to the standard enzyme-replacement therapy, administered from once per 7–14
days to 2–3 times a week in patients with severe hemophilia B, as a result of a single …
days to 2–3 times a week in patients with severe hemophilia B, as a result of a single …
Mitigating a TDP-43 proteinopathy by targeting ataxin-2 using RNA-targeting CRISPR effector proteins
MA Zeballos C, HJ Moore, TJ Smith, JE Powell… - Nature …, 2023 - nature.com
The TDP-43 proteinopathies, which include amyotrophic lateral sclerosis and frontotemporal
dementia, are a devastating group of neurodegenerative disorders that are characterized by …
dementia, are a devastating group of neurodegenerative disorders that are characterized by …
AAV engineering for improving tropism to the central nervous system
Simple Summary Adeno-associated virus (AAV) is a small, non-pathogenic, and replication-
defective virus that mainly infects primates. AAV has demonstrated great success in pre …
defective virus that mainly infects primates. AAV has demonstrated great success in pre …
The menace of severe adverse events and deaths associated with viral gene therapy and its potential solution
Over the past decade, in vivo gene replacement therapy has significantly advanced,
resulting in market approval of numerous therapeutics predominantly relying on adeno …
resulting in market approval of numerous therapeutics predominantly relying on adeno …
Optoelectronic control of cardiac rhythm: Toward shock‐free ambulatory cardioversion of atrial fibrillation
Atrial fibrillation (AF) is the most prevalent cardiac arrhythmia, progressive in nature, and
known to have a negative impact on mortality, morbidity, and quality of life. Patients requiring …
known to have a negative impact on mortality, morbidity, and quality of life. Patients requiring …
Host Cell Restriction Factors Blocking Efficient Vector Transduction: Challenges in Lentiviral and Adeno-Associated Vector Based Gene Therapies
AS Coroadinha - Cells, 2023 - mdpi.com
Gene therapy relies on the delivery of genetic material to the patient's cells in order to
provide a therapeutic treatment. Two of the currently most used and efficient delivery …
provide a therapeutic treatment. Two of the currently most used and efficient delivery …
AAV immunotoxicity: Implications in anti-HBV gene therapy
R Jacobs, MD Dogbey, N Mnyandu, K Neves, S Barth… - Microorganisms, 2023 - mdpi.com
Hepatitis B virus (HBV) has afflicted humankind for decades and there is still no treatment
that can clear the infection. The development of recombinant adeno-associated virus (rAAV) …
that can clear the infection. The development of recombinant adeno-associated virus (rAAV) …
Advances in Recombinant Adeno-Associated Virus Vectors for Neurodegenerative Diseases
L Li, L Vasan, B Kartono, K Clifford, A Attarpour… - Biomedicines, 2023 - mdpi.com
Recombinant adeno-associated virus (rAAV) vectors are gene therapy delivery tools that
offer a promising platform for the treatment of neurodegenerative diseases. Keeping up with …
offer a promising platform for the treatment of neurodegenerative diseases. Keeping up with …