Results 181 to 190 of about 55,853 (286)

Therapeutic Gene Editing: DNA Repair Pathways, Emerging Editors, and Clinical Progress

iNew Medicine, Volume 2, Issue 1, March 2026.
ABSTRACT The field of gene editing has evolved rapidly over the past decade, progressing from programmable zinc‐finger nucleases (ZFNs) and transcription activator‐like effector nucleases (TALENs) to the widespread adoption of CRISPR‐Cas systems. First‐generation editors catalyzed genome engineering by introducing targeted double‐strand breaks (DSBs ...
Li‐Kuang Tsai, Youcai Xiong, Dongqi Liu, Dongshan Yang, Jifeng Zhang, Jie Xu, Renzhi Han, Y. Eugene Chen   +7 more
wiley   +1 more source

mGem: AAV, from almost a virus to an awesome vector-or is it? [PDF]

mBio
Srivastava A.
europepmc   +1 more source

Psychological and behavioral correlates of health anxiety and other anxiety phenomena in adolescence—A cross‐sectional study in the Copenhagen Child Cohort 2000

JCPP Advances, Volume 6, Issue 1, March 2026.
Abstract Background Health anxiety (HA) is characterized by impairing worry about being or becoming seriously ill. This cross‐sectional study aimed to explore psychological and behavioral correlates of HA compared to other anxiety phenomena in adolescents, that is, with respect to depression, physical symptoms, bodily dissatisfaction, health‐related ...
Charlotte Steen Duholm, Davíð R. M. A. Højgaard, Kaare Bro Wellnitz, Eva Ørnbøl, Per Hove Thomsen, Martin Køster Rimvall, Charlotte Ulrikka Rask   +6 more
wiley   +1 more source

Acute Liver Failure With Transient Liver Steatosis Following Multiple Hits Postoperatively in a Patient With Limb-Girdle Muscular Dystrophy: A Case Report. [PDF]

Clin Case Rep
Kildal AB, Molden E, Myrseth E, Kjønås D, Oltmanns G, Goll R, Mortensen KE, Nedredal GI.   +7 more
europepmc   +1 more source

Unveiling Immune System Perturbations in Early Development Through Zebrafish Models of NADHX Repair Deficiency

Journal of Inherited Metabolic Disease, Volume 49, Issue 2, March 2026.
ABSTRACT The vital cofactors NADH and NADPH are prone to hydration, forming hydroxylated redox‐inactive derivatives (NADHX and NADPHX) in cells. These damaged metabolites are repaired by two highly conserved enzymes, an NAD(P)HX dehydratase (NAXD) and an NAD(P)HX epimerase (NAXE).
Myrto Patraskaki, Najmesadat Seyedkatouli, Lisa Schlicker, Marc O. Warmoes, Maria Lorena Cordero‐Maldonado, Ursula Heins‐Marroquin, Carole L. Linster   +6 more
wiley   +1 more source

Exploring the evidence for the use of protein biomarkers of muscular damage and disease progression in Duchenne and Becker muscular dystrophy: a systematic review and meta-analysis protocol. [PDF]

BMJ Open
Mondello S, McGill A, Lu A, Spitali P, Russell AJ, Belfiore-Oshan R, Martin PB, Duchenne Regulatory Science Consortium.   +7 more
europepmc   +1 more source

RNA‐Based Therapies for Inherited Metabolic Disorders

Journal of Inherited Metabolic Disease, Volume 49, Issue 2, March 2026.
ABSTRACT Inherited metabolic disorders (IMDs) are a diverse and complex group of genetic conditions resulting from deficiencies in enzymes, transporters, or cofactors. These deficiencies lead to metabolic dysfunction and severe clinical consequences. Despite significant progress in understanding their molecular basis, treatment options remain limited ...
Reddy Sreekanth Vootukuri, Sonam Gurung, Roopkatha Ghosh, Philippa B. Mills, Julien Baruteau, Haiyan Zhou   +5 more
wiley   +1 more source

Zebrafish as a Model Organism for Research in Rare Genetic Neuromuscular Diseases. [PDF]

Int J Mol Sci
Akyürek EE, Erba M, Dalla Barba F, Sandonà D, Sacchetto R.   +4 more
europepmc   +1 more source

Signal Peptide Engineering and Codon Optimization to Enhance α‐Gal A Activity for rAAV Gene Therapy of Fabry Disease

Journal of Inherited Metabolic Disease, Volume 49, Issue 2, March 2026.
ABSTRACT Fabry disease is an X‐linked lysosomal storage disorder caused by mutations in the GLA gene, leading to deficient α‐galactosidase A (α‐Gal A) activity and pathological accumulation of globotriaosylceramide (Gb3) and globotriaosylsphingosine (Lyso‐Gb3) in various organs.
Siwu He, Li Yang, Ke Wan, Zhaoyue Zheng, Mi Leng, Chunmei Gan, Yu Liu, Lin Xiao, Jingya Ye, Zhian Chen, Jiao Zhou, C. Alexander Valencia, Aliah Abuammah, Hoi Yee Chow, Biao Dong, Yucheng Chen   +15 more
wiley   +1 more source

Assessing Delays in Time to Diagnosis of Duchenne Muscular Dystrophy: A Survey of Current Primary Care Practices. [PDF]

Cureus
Veerapandiyan A, Hagan JF, Lipkin P, Duvuru R, Chahal J, Chahal JK, Glasner M.   +6 more
europepmc   +1 more source