LAUSR

About LAUSR
Donate
Contact Us

Follow us:

You are not signed in:

Sign Up!


   Articles with "aav gene" as a keyword



AAV gene therapy vectors in the TMJ

Sign Up to like & get
recommendations! Published in 2022 at "Clinical and Experimental Dental Research"

DOI: 10.1002/cre2.636

Abstract: The goal of this project was to evaluate the use of two adeno‐associated viral vector serotypes, adeno‐associated viral vectors (AAV)‐2 and AAV‐6, approved for and used for gene therapy in humans, for the delivery of… read more here.

Keywords: aav gene; therapy vectors; gene therapy; vectors tmj ... See more keywords

Toward improved AAV gene therapies for retinal disorders: challenges and advances.

Sign Up to like & get
recommendations! Published in 2025 at "Regenerative medicine"

DOI: 10.1080/17460751.2025.2544497

Abstract: Adeno-associated virus (AAV) vectors have transformed the landscape of in vivo gene therapy, with retinal diseases emerging as a major area of progress. The eye offers unique advantages as a therapeutic target: it is accessible,… read more here.

Keywords: gene therapies; toward improved; gene therapy; aav gene ... See more keywords
Photo from wikipedia

Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?

Sign Up to like & get
recommendations! Published in 2021 at "Expert opinion on orphan drugs"

DOI: 10.1080/21678707.2021.1882300

Abstract: ABSTRACT Introduction: Choroideremia is an X-linked inherited retinal degeneration resulting from mutations in the CHM gene, encoding Rab escort protein-1 (REP1), a protein regulating intracellular vesicular transport. Loss-of-function mutations in CHM lead to progressive loss… read more here.

Keywords: choroideremia; subretinal aav; option choroideremia; gene ... See more keywords

Prophylaxis CTLA4-ig reduces anti-AAV neutralizing antibodies in AAV gene therapy for hemophilia

Sign Up to like & get
recommendations! Published in 2025 at "Blood"

DOI: 10.1182/blood-2025-1293

Abstract: Objective: This study aimed to investigate the inhibitory effect of CTLA4-Ig on anti-AAV neutralizing antibody (Nab) development after AAV gene therapy in hemophilia patients, providing an immunomodulatory strategy for potential re-treatment. Methods: Key immune targets… read more here.

Keywords: ctla4; anti aav; gene therapy; aav gene ... See more keywords
Photo from wikipedia

Adeno-associated viral integration profiles in human liver biopsies following Hemophilia A gene therapy

Sign Up to like & get
recommendations! Published in 2025 at "Blood"

DOI: 10.1182/blood-2025-4315

Abstract: Introduction: Adeno-associated viral (AAV) gene therapy has received approvals for the treatment of severe hemophilia A. Preclinical studies on the natural history of AAV have shown that, although most vectors persist as extrachromosomal episomal forms,… read more here.

Keywords: integration; gene therapy; aav gene; evidence ... See more keywords

1347 Using Living Human Glioblastoma Samples to Develop Tumor-Targeted AAV Gene Therapy Vectors

Sign Up to like & get
recommendations! Published in 2025 at "Neurosurgery"

DOI: 10.1227/neu.0000000000003360_1347

Abstract: INTRODUCTION: Glioblastoma multiforme is the most common primary intracranial malignant brain tumor in adults, known for its aggressive behavior and poor prognosis. Several therapeutic approaches have been described; unfortunately, no treatment alternatives have substantially impacted… read more here.

Keywords: aav gene; tumor; glioblastoma; gene therapy ... See more keywords

Creating an arsenal of Adeno-associated virus (AAV) gene delivery stealth vehicles

Sign Up to like & get
recommendations! Published in 2018 at "PLoS Pathogens"

DOI: 10.1371/journal.ppat.1006929

Abstract: The Adeno-associated virus (AAV) gene delivery system is ushering in a new and exciting era in the United States; following the first approved gene therapy (Glybera) in Europe, the FDA has approved a second therapy,… read more here.

Keywords: gene; aav gene; gene delivery; adeno associated ... See more keywords

Organoids and microphysiological systems: Promising models for accelerating AAV gene therapy studies

Sign Up to like & get
recommendations! Published in 2022 at "Frontiers in Immunology"

DOI: 10.3389/fimmu.2022.1011143

Abstract: The FDA has predicted that at least 10-20 gene therapy products will be approved by 2025. The surge in the development of such therapies can be attributed to the advent of safe and effective gene… read more here.

Keywords: aav gene; organoids microphysiological; gene therapy; microphysiological systems ... See more keywords

Immunogenicity and toxicity of AAV gene therapy

Sign Up to like & get
recommendations! Published in 2022 at "Frontiers in Immunology"

DOI: 10.3389/fimmu.2022.975803

Abstract: Gene transfer using adeno-associated viral (AAV) vectors has made tremendous progress in the last decade and has achieved cures of debilitating diseases such as hemophilia A and B. Nevertheless, progress is still being hampered by… read more here.

Keywords: toxicity aav; aav gene; aav vectors; gene ... See more keywords