Results 171 to 180 of about 39,202 (243)

Health care costs of home care enzyme replacement therapy for patients with lysosomal storage diseases in Germany. [PDF]

Orphanet J Rare Dis
Heinrich R, Claus F, Schoenfelder T.
europepmc   +1 more source

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

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

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

Comparative Transcriptional Profiling of Key Macrophage and Fibroblast Subpopulations in Rheumatoid Arthritis–Associated Lung Disease

Arthritis &Rheumatology, EarlyView.
Objective Rheumatoid arthritis (RA) often involves extra‐articular complications, including interstitial lung disease (ILD) and/or pulmonary nodules. Transcriptomic profiling of lung tissue provides the opportunity to directly assess cell‐specific gene expression and corresponding pathway activation in different types of rheumatoid lung disease ...
Tracy Tabib, Ogechukwu Ezenwa, Joshua Sciurba, Eleanor B. Valenzi, John Sembrat, Brittany Cody, Jishnu Das, Robert Lafyatis, Dana P. Ascherman   +8 more
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

Purification of Advanced Therapeutics by Flow‐Through Pseudo‐Affinity Chromatography: Bi‐Specific mABs, Fc Fusion Proteins, and Adeno‐Associated Viruses

Biotechnology and Bioengineering, EarlyView.
ABSTRACT Modern biopharmaceutical manufacturing requires purification platforms capable of processing structurally and functionally diverse products while addressing the challenge of removing persistent and high‐risk host cell proteins (HCPs).
Wenning Chu, Eduardo Barbieri, Arianna Minzoni, Shriarjun Shastry, Jacob P. LeBarre, Qingbo Shu, Taufika Williams, Xuanwen Li, Divya Chandra, Xuankuo Xu, Jerome Bill, David Roush, Joseph Taris, Patrick Gilbert, Andre Kiesewetter, Oliver Rammo, Michael Schulte, Christopher Major, Trina Mouchahoir, Steven Cramer, Cristiana Boi, Ruben G. Carbonell, Abraham Lenhoff, Stefano Menegatti   +23 more
wiley   +1 more source

Harnessing ferroptosis from multilayer defense networks to nanoplatforms for specific cancer therapy

BMEMat, EarlyView.
Nanomaterials target metabolically‐regulated ferroptosis for cancer therapy. Iron‐based or alternative nanoplatforms integrate ferroptosis with chemotherapy, immunotherapy, or radiotherapy. They enable stimulus‐responsive therapies (photothermal, photodynamic, sonodynamic) activated by near‐infrared, light, or ultrasound, achieving potent synergistic ...
Xinyue Xu, Xuejian Wang, Wenyan Jiang, Wan Hairul Anuar Kamaruddin, Chunyan Fang, Pengfei Zhang   +5 more
wiley   +1 more source