Results 161 to 170 of about 131,722 (303)

Clinical‐Grade Human Induced Pluripotent Stem Cell‐Derived Neural Precursor Cells Restore Motor Function and Preserve Striatal Integrity in a Quinolinic Acid‐Lesioned Rat Model of Huntington's Disease

Cell Proliferation, EarlyView.
Clinical‐grade HLA‐homozygous iPSC‐derived neural precursor cells restore motor function, rebuild striatal circuitry and reduce neuroinflammation in QA‐lesioned rats. These findings demonstrate robust neuronal replacement and microenvironment modulation, supporting their potential as a regenerative therapy for Huntington's disease.
Hyeonjoong Jeon, Il‐Shin Lee, Sanghun Lee, Hyo Chang Park, Beomsoo Kim, Hyun Sook Kim, Jihwan Song   +6 more
wiley   +1 more source

An LNA gapmer antisense oligonucleotide inhibitor of human NNMT. [PDF]

Mol Ther Nucleic Acids
Chen S, Poudel BH, Veedu RN.
europepmc   +1 more source

Garadacimab for the long‐term prophylaxis of hereditary angioedema

JDDG: Journal der Deutschen Dermatologischen Gesellschaft, EarlyView.
Summary Hereditary angioedema (HAE), a rare and debilitating disease characterized by recurrent and spontaneous attacks of tissue swelling, has a high unmet therapeutic need, with many patients experiencing insufficient disease control with current prophylactic treatments.
Emel Aygören‐Pürsün, Regina Treudler, Petra Staubach, Inmaculada Martinez Saguer, Thomas Linhoff, Markus Magerl   +5 more
wiley   +1 more source

Systemic distribution of tricyclo-DNA antisense oligonucleotide following intratracheal instillation in the mouse. [PDF]

NAR Mol Med
Laurent J, Deramaudt TB, Cloix R, Robin V, Beley C, Garcia L, Bonay M.   +6 more
europepmc   +1 more source

Algorithms in Allergy: Hereditary Angioedema

Allergy, EarlyView.
Konrad Bork, Markus Magerl, Emel Aygören‐Pürsün, Henriette Farkas   +3 more
wiley   +1 more source

Regulation of PHOX2B gene expression by the long non‐coding natural antisense RNA PHOX2B‐AS1

The FEBS Journal, EarlyView.
PHOX2B is a transcription factor essential for autonomic nervous system development. We identify and characterize PHOX2B‐AS1, a human long non‐coding antisense transcript at the PHOX2B locus, along with its murine counterpart. Our findings reveal bidirectional transcription and reciprocal regulation: PHOX2B activates PHOX2B‐AS1, whereas PHOX2B‐AS1 ...
Simona Di Lascio, Ana Lucia Cuadros Gamboa, Martina Bertocchi, Filippo Chiesa, Francesca Cargnin, Ettore Mosca, Paride Pelucchi, Viviana Tritto, Stefania Corti, Isabella Ceccherini, Paola Riva, Roberta Benfante, Diego Fornasari   +12 more
wiley   +1 more source

Phosphorothioate antisense oligonucleotide induced innate immune activation is attenuated by tryptophan oxidation products. [PDF]

Nucleic Acids Res
Pytte J, Saldana L, Zhang W, Skourti-Stathaki K, Crooke ST.   +4 more
europepmc   +1 more source

Antisense Oligonucleotides [PDF]

, 2020
openaire   +1 more source

Investigating transthyretin variants H88R and I107V in amyloid priming: From destabilization to complete dissociation

The FEBS Journal, EarlyView.
Investigated mutations in transthyretin (TTR) disrupt the F87‐centered hydrophobic core that stabilizes its tetrameric structure. The mild I107V mutation weakens inter‐chain packing, while H88R fully abolishes tetramer formation, yielding a monomeric, aggregation‐prone form. Structural, biophysical, and computational analyses reveal that both mutations
István L. Bódy, Zsolt Fazekas, Nóra Wágner, Anna J. Kiss‐Szemán, Veronika Harmat, Zoltán Pozsonyi, Dóra K. Menyhárd, András Perczel   +7 more
wiley   +1 more source

Integrating transcriptomics and proteomics to assess antisense oligonucleotide safety and efficacy: a time-resolved approach. [PDF]

NAR Mol Med
van Leeuwen D, Cipullo M, Williams EC, Iannetta A, Chu B, Wang J, Hamza G, Crysnanto D, Guzzi N, Tan JY, Miele E, Sawarkar R, Mohorianu I, Prill S, Andersson P.   +14 more
europepmc   +1 more source