Results 121 to 130 of about 2,583,576 (275)
Biotechnology and Bioengineering, Volume 122, Issue 5, Page 1067-1086, May 2025.ABSTRACT Recombinant adeno‐associated virus (rAAV) has emerged as an attractive gene delivery vector platform to treat both rare and pervasive diseases. With more and more rAAV‐based therapies entering late‐stage clinical trials and commercialization, there is an increasing pressure on the rAAV manufacturing process to accelerate drug development ...
Julius Klemens Lorek +2 more
wiley +1 more source
Hemophilia B in a Pair of Monozygotic Negro Twins [PDF]
, 1964 Israel Roisenberg
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A Toolkit for Healthcare Transition for Adolescents With Classical Myeloproliferative Neoplasms
Pediatric Blood &Cancer, Volume 72, Issue 5, May 2025.ABSTRACT Classical myeloproliferative neoplasms (MPNs) are being identified more frequently in adolescents. There is no guidance on the healthcare transition of young MPN patients from pediatric to adult medicine. Therefore, we convened an international panel of experts in both pediatric and adult MPN care to develop three tools to facilitate high ...
Nicole Kucine +8 more
wiley +1 more source
Advanced Science, Volume 12, Issue 14, April 10, 2025.A new CdCS rat model is developed using CRISPR‐Cas9 technology. It shows impairments in cognitive and social behaviors, neuronal dendrite abnormalities in the hippocampus and mPFC, and inflammatory and immune processes in mPFC brain cells. Early AAV‐Ctnnd2 gene therapy enhances cognitive function in CdCS rats.
Jingjing Shen +11 more
wiley +1 more source
Clinical Pharmacology &Therapeutics, Volume 117, Issue 4, Page 1098-1105, April 2025.In the EU, conditional marketing authorization is a pragmatic tool for early approval of a medicine that fulfills an unmet medical need. In the pharmaceutical legislation, an unmet medical need means that a condition lacks a satisfactory method for diagnosis, prevention, or treatment.
Florian Lasch +2 more
wiley +1 more source
Pennsylvania state-wide hemophilia program: summary of immediate reactions with the use of factor VIII and factor IX concentrate [PDF]
, 1979 David Prager +8 more
openalex +1 more source
Diverse potential of chimeric antigen receptor‐engineered cell therapy: Beyond cancer
Clinical and Translational Medicine, Volume 15, Issue 4, April 2025.1. In theory, CAR‐engineering therapy can achieve therapeutic effects by targeting cells expressing specific antigens and thereby eliminating or regulating disease‐related cell subpopulations. 2. Although CAR‐engineering therapy is currently only approved for the treatment of cancer, it has also shown potential in non‐tumour diseases. 3.
Lvying Wu, Lingfeng Zhu, Jin Chen
wiley +1 more source
Immune Checkpoint Molecules: A Review on Pathways and Immunotherapy Implications
Immunity, Inflammation and Disease, Volume 13, Issue 4, April 2025.Immune checkpoint molecules are today's leading targets for immunotherapy, especially in the field of cancer. This review summarizes the most important and recent advances in this field and in different types of cancer. ABSTRACT Background Today, treating cancer patients with monoclonal antibodies (mAbs), by targeting immune checkpoints, is one of the ...
Erfan Rezazadeh‐Gavgani +4 more
wiley +1 more source