Results 131 to 140 of about 72,117 (254)

Optimizing Oligonucleotide Therapeutics: A Model-Informed Drug Development Perspective. [PDF]

Clin Transl Sci
Yuan Y, Sharma V, Bhattaram VA, Pan X, Earp J, Wang Y, Liu J, Zhu H.   +7 more
europepmc   +1 more source

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

Recent developments in the delivery of peptide nucleic acids (PNAs). [PDF]

RSC Chem Biol
Sarkar S, Murthy N.
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

Antisense oligonucleotides reverse SPTLC1-related hereditary sensory neuropathy in a mouse model. [PDF]

Brain
Meng J, Ma S, Lone MA, Lam HW, Zhang Q, Cheng S, Mackie S, Graham E, Kedzior H, Demetriou C, Ziak N, Andreoli L, Beggs S, Koch S, Clark AJ, Bennett DL, Hornemann T, Muntoni F, Reilly MM, Zhou H.   +19 more
europepmc   +1 more source

Comparative Effects of Emerging Lp(a)‐Lowering Agents and PCSK9‐Directed Therapies on Lipoprotein(a): A Network Meta‐Analysis of Randomised Clinical Trials

Diabetes, Obesity and Metabolism, EarlyView.
ABSTRACT Aims Elevated lipoprotein(a) [Lp(a)] is a genetic ASCVD risk factor that often persists despite intensive LDL‐C lowering. We compared the efficacy and safety of emerging Lp(a)‐targeted therapies (siRNAs, antisense oligonucleotides and an oral assembly inhibitor) with PCSK9‐directed therapies.
Jihad Abu Zayed, Nada A. Al‐Awamleh, Mahmoud Hamad, Mohammad Ibrahim Mahmoud Hdaib, Hazem Ayesh   +4 more
wiley   +1 more source

Direct targeting of C9ORF72 repeat RNA with fluorinated antisense oligonucleotides. [PDF]

Nucleic Acids Res
Barber HM, Parasrampuria MA, Jurado-Arjona J, Gamir-Morralla A, Berninger B, González C, Gagnon KT, Damha MJ, Garavís M.   +8 more
europepmc   +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

RNA-Based Therapies for Hypercholesterolemia and Coronary Artery Disease. [PDF]

Cureus
Lopez Mendoza MJ, Contreras Figueroa N, Valle Mena MJ, Ulloa A, Jiron Vindas J, Salazar Estrada MA.   +5 more
europepmc   +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