Results 201 to 210 of about 3,524,002 (263)

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

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

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

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

Nanomedicine‐driven innovations in postoperative cancer immunotherapy: Remodeling tumor microenvironment and precision delivery strategies

BMEMat, EarlyView.
Nanomedicine‐Driven Strategies for Suppressing Postoperative Tumor Recurrence. This schematic illustrates three pivotal nanomedical strategies that synergistically counteract recurrence by targeting residual disease and the immunosuppressive tumor microenvironment within the surgical cavity.
Lisong Pang, Zhuomin Wang, Zhenhua Jin, Ahmed H. Ghonaim, Hengcheng Zhang, Xinyu You, Jinzhuo Ning, Jiaqing Cao, Yang Liu   +8 more
wiley   +1 more source

Patient and family experiences of lysosomal storage diseases in Canada: A qualitative interview study. [PDF]

JIMD Rep
Awada N, Holcik M.
europepmc   +1 more source

Bridging the translational gap in clinical nanomedicine: From rational design to clinical reality

BMEMat, EarlyView.
Nanotechnology offers a multi‐faceted approach to modern healthcare, encompassing high‐sensitivity diagnostic imaging, innovative vaccine delivery, and targeted therapeutics. This review explores how these nanomedicine applications address critical challenges in cancer recurrence and the treatment of neurologic and respiratory diseases to combat rising
Ahmed H. Ghonaim, Marwa Ahmed El‐Naka, Wedad M. Nageeb, Atta Ullah Khan, Noura M. Seleem, Aliaa Abdelghafar, Doaa Zamel, Yang Liu, Qigai He, Wentao Li   +9 more
wiley   +1 more source

Probiotic spore‐based bioresponsive drug delivery for reversing inflammatory bowel disease and its associated anxiety and depression

BMEMat, EarlyView.
A probiotic spore‐based drug delivery system (BCS‐Que@ApNPs) provides excellent targeted delivery, bioresponsive drug release, and long‐term retention (over 48 h), making it suitable for oral treatment of inflammatory bowel disease and its associated anxiety and depression.
Qingling Song, Beiyang Xue, Yao Li, Chuangxi Zhou, Junfei Yang, Yunfei Li, Lingwen Ding, Yun Zhang, Hongjuan Zhao, Lei Wang   +9 more
wiley   +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