Results 141 to 150 of about 37,661 (243)

Neurological manifestations of lysosomal storage diseases. [PDF]

Ann Med Surg (Lond)
Elendu C, Babawale EA, Babarinde FO, Babatunde OD, Chukwu C, Chiegboka SF, Shode OP, Ngozi-Ibeh JK, Njoku A, Ikokwu MN, Kaka GU, Hassan JI, Fatungase OO, Osifodunrin T, Udoeze CA, Ikeji VI.   +15 more
europepmc   +1 more source

Advancing the Research and Development of Enzyme Replacement Therapies for Lysosomal Storage Diseases. [PDF]

GEN Biotechnol, 2022
Puhl AC, Ekins S.
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

Lysosomal Storage Diseases—Regulating Neurodegeneration

Journal of Experimental Neuroscience, 2016
Rob U. Onyenwoke, Jay E. Brenman
doaj  

Feasibility and efficacy of the forced oscillation technique in patients with lysosomal storage diseases. [PDF]

Sci Rep
Alblooshi A, Zaabi NA, Albadi G, Al-Jasmi FA.   +3 more
europepmc   +1 more source

Secondary Mitochondrial Dysfunction as a Cause of Neurodegenerative Dysfunction in Lysosomal Storage Diseases and an Overview of Potential Therapies. [PDF]

Int J Mol Sci, 2022
Stepien KM, Cufflin N, Donald A, Jones S, Church H, Hargreaves IP.   +5 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

Extracellular Vesicles as Tools for Crossing the Blood-Brain Barrier to Treat Lysosomal Storage Diseases. [PDF]

Life (Basel)
Lerussi G, Villagrasa-Araya V, Moltó-Abad M, Del Toro M, Pintos-Morell G, Seras-Franzoso J, Abasolo I.   +6 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

Prenatal Delivery of Enzyme Replacement Therapy to Fetuses Affected by Early-Onset Lysosomal Storage Diseases. [PDF]

Am J Med Genet C Semin Med Genet
Borges B, Canepa E, Chang IJ, Herzeg A, Lianoglou B, Kishnani PS, Harmatz P, MacKenzie TC, Cohen JL.   +8 more
europepmc   +1 more source