Results 51 to 60 of about 144,727 (384)
AAV: An Overview of Unanswered Questions [PDF]
Human Gene Therapy, 2017 AAV has been studied for 55 years and has been developed as a vector for about 35 years. By now, there is a fairly good idea of the dimensions of what would be useful to know to employ AAV optimally as a vector, but there are still many unanswered questions within the system. As with all biological systems, each good experiment raises further questions
Nicholas Muzyczka, Kenneth I. Berns
openaire +3 more sources
Adeno-Associated Virus (AAV) - Based Gene Therapies for Retinal Diseases: Where are We?
The Application of Clinical Genetics, 2023 Divya Ail, Hugo Malki, Emilia A Zin, Deniz Dalkara Sorbonne Université, INSERM, CNRS, Department of Therapeutics, Institut de la Vision, Paris, 75012, FranceCorrespondence: Divya Ail, Department of Therapeutics, Institut de la Vision, 17 Rue Moreau ...
Ail D, Malki H, Zin EA, Dalkara D
doaj
Emerging Concepts and Challenges in Rheumatoid Arthritis Gene Therapy
Biomedicines, 2020 Rheumatoid arthritis (RA) is a systemic inflammatory joint disease affecting about 1% of the population worldwide. Current treatment approaches do not ensure a cure for every patient.
Andrei A. Deviatkin +6 more
doaj +1 more source
Frontiers in Bioengineering and Biotechnology, 2021 Of the adeno-associated viruses (AAVs), AAV9 is known for its capability to cross the blood–brain barrier (BBB) and can, therefore, be used as a noninvasive method to target the central nervous system.
Kimberly L. Pietersz +9 more
doaj +1 more source
Thorough molecular configuration analysis of noncanonical AAV genomes in AAV vector preparations
Molecular Therapy: Methods & Clinical DevelopmentThe unique palindromic inverted terminal repeats (ITRs) and single-stranded nature of adeno-associated virus (AAV) DNA are major hurdles to current sequencing technologies.
Junping Zhang +7 more
doaj +4 more sources
LDLR-Gene therapy for familial hypercholesterolaemia: Problems, progress, and perspectives [PDF]
, 2010 Coronary artery diseases (CAD) inflict a heavy economical and social burden on most populations and contribute significantly to their morbidity and mortality rates.
Al-Allaf, FA +5 more
core +3 more sources
Protective Effects of Human and Mouse Soluble Scavenger-Like CD6 Lymphocyte Receptor in a Lethal Model of Polymicrobial Sepsis [PDF]
, 2016 Sepsis still constitutes an unmet clinical need, which could benefit from novel adjunctive strategies to conventional antibiotic therapy. The soluble form of the scavenger-like human CD6 lymphocyte receptor (shCD6) binds to key pathogenic components from
Aranda, Fernando +9 more
core +1 more source
Science Advances, 2020 Self-complementary AAV-packaged CRISPR-Cas9 genome editing components rescue Duchenne muscular dystrophy. Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disease caused by mutations in the dystrophin gene (DMD).
Yu Zhang +10 more
semanticscholar +1 more source
Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus. [PDF]
, 2013 Usher 1 patients are born profoundly deaf and then develop retinal degeneration. Thus they are readily identified before the onset of retinal degeneration, making gene therapy a viable strategy to prevent their blindness.
Boye, S +8 more
core +1 more source
Evolving AAV-delivered therapeutics towards ultimate cures
Journal of molecular medicine, 2021 Gene therapy has entered a new era after decades-long efforts, where the recombinant adeno-associated virus (AAV) has stood out as the most potent vector for in vivo gene transfer and demonstrated excellent efficacy and safety profiles in numerous ...
Xiangjun He +4 more
semanticscholar +1 more source