Results 161 to 170 of about 239,401 (300)

Bringing Gene Therapy Into Real World Clinical Practice

Haemophilia, EarlyView.
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, Pratima Chowdary, Margareth C. Ozelo   +2 more
wiley   +1 more source

What is driving the resurgence and persistence of vaccine-targeted pneumococcal serotypes?-Serotype 19F as the paradigm. [PDF]

PLoS Pathog
Thindwa D, Carcamo PM, Dagan R, Weinberger DM.   +3 more
europepmc   +1 more source

Human Rhinovirus A16 Infection of Mast Cells Promotes T2 and Non‐T2 Immune Responses That Are Modulated by the Airway Epithelium

Allergy, EarlyView.
Ying Lai, Ryan C. Murphy, Matthew Liu, Maciej W. Czarnecki, Dane Grosvenor, Kimberly A. Dill‐McFarland, Adrian M. Piliponsky, Jason S. Debley, Matthew C. Altman, Teal S. Hallstrand   +9 more
wiley   +1 more source

Gene Editing for Haemophilia—The Next Frontier

Haemophilia, EarlyView.
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, Gregory A. Newby, Sundar Selvaraj, Steven W. Pipe   +3 more
wiley   +1 more source

Treatment With Valoctocogene Roxaparvovec in a Patient With Severe Hemophilia A Led to Sustained Normal FVIII Levels

Haemophilia, EarlyView.
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, Behnaz Pezeshkpoor, Claudia Klein, Thilo Albert, Philipp Lutz, Christian Strassburg, Ulrich Spengler, Georg Goldmann, Johannes Oldenburg   +8 more
wiley   +1 more source

Outbreak of Dengue Virus Serotype 3, Republic of the Marshall Islands, 2019-2021. [PDF]

Emerg Infect Dis
León TM, McCready J, Chutaro E, McAllister J, Solomon B, Hapairai LK.   +5 more
europepmc   +1 more source

Will Extended‐Valency Pneumococcal Conjugate Vaccines Offer Enhanced Coverage Against Invasive Pneumococcal Disease for At‐Risk Children?

Acta Paediatrica, EarlyView.
Robert Cohen, Corinne Levy, Naim Ouldali, Stéphane Béchet, Aude Estivaux, André Birgy, Isabelle Hau, IPD Study Group, Emmanuelle Varon   +8 more
wiley   +1 more source

Final Analysis of the Phase 1/2 Trial of Valoctocogene Roxaparvovec for Severe Haemophilia A

Haemophilia, EarlyView.
ABSTRACT Introduction Valoctocogene roxaparvovec is an adeno‐associated virus vector serotype 5 (AAV5)‐mediated gene therapy for severe haemophilia A (HA). Aim Report the final safety and efficacy results of the phase 1/2 trial of valoctocogene roxaparvovec. Methods An open‐label phase 1/2 trial (NCT02576795) enrolled adult males with severe HA (factor
Priyanka Raheja, Savita Rangarajan, Will Lester, Bella Madan, Glenn F. Pierce, Emily Symington, Carolyn Millar, Dane Osmond, Mingjin Li, Konstantia‐Maria Chavele   +9 more
wiley   +1 more source

Development and Validation of Sixplexed Opsonophagocytic Killing Assay for Serotype-Specific Functional Pneumococcal Antibody Measurement. [PDF]

Vaccines (Basel)
Jang AY, Ji HJ, Choi YJ, Nham E, Yoon JG, Choi MJ, Noh JY, Cheong HJ, Seo HS, Song JY.   +9 more
europepmc   +1 more source

Considerations for the Use of AAV‐based Gene Therapy in HIV‐Positive Individuals With Haemophilia

Haemophilia, EarlyView.
ABSTRACT Introduction There is a high prevalence of human immunodeficiency virus (HIV) infection among the haemophilia community due to treatment in the 1970s and 1980s with contaminated clotting factor. Lifelong treatment regimens for haemophilia and HIV are burdensome alone and pose a particular challenge for individuals living with both conditions ...
Jürgen K. Rockstroh, Julia C. Stingl, Marco Stadler, Anja Reichert, Heiner Wedemeyer, Johannes Oldenburg   +5 more
wiley   +1 more source