Next‐generation sequencing in newborn screening: Current status, challenges, and future perspectives
Newborn screening shifts from biochemical to genetic methods. Global exploration is promising but requires overcoming challenges and international collaboration to optimize implementation. ABSTRACT Newborn screening (NBS) is a key public health intervention that improves children's health outcomes by enabling precise intervention through the early ...
Zhelan Huang, Wenhao Zhou
wiley +1 more source
Fetal bone engraftment reconstitutes the immune system in pigs with severe combined immunodeficiency. [PDF]
Monarch K +16 more
europepmc +1 more source
Plants, like humans, have a microbiome that helps them grow, defend themselves against pathogens, acquire nutrients, and protect themselves against environmental stresses. The microbiome of tomatoes, a staple crop grown worldwide, could be utilized not only to reduce fertilizer and pesticide applications, but also to clean up harmful pollutants ...
Sean Lindert +3 more
wiley +1 more source
Multi-Year Registry Study of Elapegademase Treatment in Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Requiring Enzyme Replacement Therapy. [PDF]
Dorsey MJ +19 more
europepmc +1 more source
Disease Progression Mathematical Modeling With a Case Study on Hepatitis B Virus Infection
ABSTRACT Chronic Hepatitis B presents a significant health and socioeconomic burden. The risk of hepatocellular carcinoma remains elevated although treatments are available. Achieving an optimal treatment regimen necessitates a deep comprehension of the dynamic relationship between the virus and its host across disease states.
Clémence Boivin‐Champeaux +6 more
wiley +1 more source
Comprehensive newborn screening for severe combined immunodeficiency, X-linked agammaglobulinemia, and spinal muscular atrophy: the Chinese experience. [PDF]
Chen C +8 more
europepmc +1 more source
ABSTRACT Lung cancer remains the leading cause of cancer‐related deaths worldwide, yet immune checkpoint blockade (ICB) is often compromised by an immunosuppressive tumor microenvironment (TME) with acidosis, hypoxia, and poor T cell infiltration. Here, we develop a TME‐responsive nanoplatform (MgH2@Lip/PD‐L1) that couples hydrogen gas therapy with PD ...
Xuyu Gu +5 more
wiley +1 more source
Validation on the First-Tier Fully Automated High-Throughput <i>SMN1</i>, <i>SMN2</i>, TREC, and <i>RPP30</i> Quantification by Quadruplex Droplet Digital PCR for Newborn Screening for Spinal Muscular Atrophy and Severe Combined Immunodeficiency. [PDF]
Mak CM +9 more
europepmc +1 more source
This review highlights recent advances in nanomedicine that enhance chimeric antigen receptor (CAR) T cell therapy against solid tumors. It discusses key barriers to CAR T cell efficacy and outlines nanotechnology‐based strategies to improve tumor targeting, remodel the immunosuppressive microenvironment, and integrate nanomaterials into CAR T cell ...
Stephen O’Rourke +3 more
wiley +1 more source

