Results 171 to 180 of about 38,108 (242)

The Liver and Lysosomal Storage Diseases: From Pathophysiology to Clinical Presentation, Diagnostics, and Treatment. [PDF]

Diagnostics (Basel)
Lipiński P, Tylki-Szymańska A.
europepmc   +1 more source

Micro‐ and nanoplastics in gastrointestinal disorders: Mapping the translational gap between exposure, biomonitoring, and pathogenic mechanisms

BMEMat, EarlyView.
This review highlights critical “translational gaps” in understanding micro‐ and nanoplastics impacts on gastrointestinal diseases, bridging exposure, detecting, and mechanistic toxicology. Abstract Micro‐ and nanoplastics (MNPs) have become pervasive environmental contaminants with increasing evidence linking them to adverse health outcomes.
Zhenli Diao, Yujie Luo, Chuanda Zhu, Yanwei Hu   +3 more
wiley   +1 more source

A PIKfyve modulator combined with an integrated stress response inhibitor to treat lysosomal storage diseases. [PDF]

Proc Natl Acad Sci U S A
Hou WC, Massey LA, Rhoades D, Wu Y, Ren W, Frank C, Overkleeft HS, Kelly JW.   +7 more
europepmc   +1 more source

Near‐infrared light‐activated aptamer‐based nanoprobe for spatiotemporally resolved detection of L‐lactate in colorectal cancer

BMEMat, EarlyView.
A near‐infrared (NIR) light‐activated “lock‐key” nanodevice is reported for spatiotemporally resolved detection of intratumoral L‐lactate. The system stays silent until deep‐penetrating NIR light remotely unlocks its function, allowing for precise sensing within tumors.
Siyu Pan, Xuanyi Lu, Wenjie Yang, Yunhui Peng, Lingfu Deng, Shuai Zha, Xueyang Fang, Guochen Bao, Lijun Jiang   +8 more
wiley   +1 more source

Hematopoietic stem cell gene therapy of neurometabolic lysosomal storage diseases.

Brain Dev
Consiglieri G, Tucci F, Bernardo ME.
europepmc   +1 more source

Diagnostic networks for IEM in Brazil: report on 10 years of activity of the LSD Brazil Network, helping Brazil and Latin America to identify patients with lysosomal storage diseases. [PDF]

J Community Genet
Trapp FB, Riegel M, Michelin-Tirelli K, da Silva LAL, Iop GD, Bender-Pasetto F, Sebastião FM, da Silva Moraes I, Lopes FF, Dos Santos ORT, da Rosa Pereira F, Silva LP, Luiz AA, da Silva CA, Lucas KDP, Netto ABO, Brasil CS, Leistner S, Brusius-Facchin AC, Deon M, Civallero GES, Giugliani R.   +21 more
europepmc   +1 more source

Status and future of recombinant adeno‐associated virus vector manufacturing

Biotechnology Progress, EarlyView.
Abstract Sixty years of adeno‐associated virus (AAV) research illustrates a trajectory marked by basic science exploration, iterative innovation, persistent challenges, a number of clinical setbacks, as well as commercial therapeutic triumphs. This continual evolution has led to recombinant AAV (rAAV) becoming a cornerstone of modern gene therapy ...
Frank Agbogbo, David Dismuke
wiley   +1 more source

Systematic Review of Genetic Substrate Reduction Therapy in Lysosomal Storage Diseases: Opportunities, Challenges and Delivery Systems. [PDF]

BioDrugs
Beraza-Millor M, Rodríguez-Castejón J, Del Pozo-Rodríguez A, Rodríguez-Gascón A, Solinís MÁ.   +4 more
europepmc   +1 more source

Organelle‐Resolved Tetrazine‐trans‐Cyclooctene Click Chemistry for Cargo Delivery and Release

Chemistry – A European Journal, EarlyView.
Bioorthogonal click chemistry tools provide a means for specific intracellular conjugation of molecules. In this study, we used reactive tetrazine (Tz) and TCO moieties for labeling of organelles and organelle‐specific delivery and activation of doxorubicin prodrugs.
Oleh Durydivka, Marek Chovanec, Rastislav Dzijak, Michal Rahm, Jachym Hrusak, Paul E. Reyes‐Gutierrez, Milan Vrabel   +6 more
wiley   +1 more source