Results 191 to 200 of about 192,156 (336)
What is driving the resurgence and persistence of vaccine-targeted pneumococcal serotypes?-Serotype 19F as the paradigm. [PDF]
Thindwa D +3 more
europepmc +1 more source
Bringing Gene Therapy Into Real World Clinical Practice
ABSTRACT Introduction Adeno‐associated virus (AAV)‐based gene therapy for haemophilia has shifted therapeutic paradigms by enabling hepatic gene transfer, restoring endogenous clotting factor expression, and reducing reliance on conventional prophylactic treatments. Two products, valoctocogene roxaparvovec (haemophilia A) and etranacogene dezaparvovec (
Wolfgang Miesbach +2 more
wiley +1 more source
Genomic characterization of mobile genetic elements associated with antimicrobial resistance in <i>Streptococcus pneumoniae</i> from Australia. [PDF]
Sunmonu GT +5 more
europepmc +1 more source
Gene Editing for Haemophilia—The Next Frontier
ABSTRACT The recently approved haemophilia A and B gene therapies via adeno‐associated virus (AAV) showed a promising therapeutic response after a single injection, but there are still limitations, including the potential loss of transgene expression and restriction in adults.
Mirko Pinotti +3 more
wiley +1 more source
Development and Validation of Sixplexed Opsonophagocytic Killing Assay for Serotype-Specific Functional Pneumococcal Antibody Measurement. [PDF]
Jang AY +9 more
europepmc +1 more source
ABSTRACT Introduction Valoctocogene roxaparvovec, an adeno‐associated virus (AAV)‐based gene therapy, enables endogenous factor VIII (FVIII) expression in patients with severe hemophilia A without the need for regular FVIII infusions. Long‐term follow‐up assesses durability, safety, and immune‐related challenges following gene therapy.
Kerstin Herbst +8 more
wiley +1 more source

