Results 61 to 70 of about 1,115 (145)

Clinical Features and Outcomes of Acute Intermittent Porphyria Presenting With Acute Quadriparesis: A Case Series and Follow‐Up Study

European Journal of Neurology, Volume 32, Issue 6, June 2025.
ABSTRACT Introduction A retrospective case series of acute intermittent porphyria (AIP) presenting with acute quadriparesis is described with a focus on patterns of neuropathy and nerve conduction study findings. Methods Six patients with acute polyneuropathy were diagnosed with AIP on the basis of characteristic clinical findings, urine ...
Reem M. Alhammad, Rema A. Almutawa, Abdulrahman S. Ali, Nuha M. Alkhawajah, Hana M. Albulaihe, Afag M. Osman, Mohammed A. AlSheef, Alaa A. Alsayed, Raashda A. Sulaiman, Khalid J. Alharbi   +9 more
wiley   +1 more source

Principle, application and challenges of development siRNA-based therapeutics against bacterial and viral infections: a comprehensive review

Frontiers in Microbiology
While significant progress has been made in understanding and applying gene silencing mechanisms and the treatment of human diseases, there have been still several obstacles in therapeutic use. For the first time, ONPATTRO, as the first small interfering
Hamid Motamedi, Hamid Motamedi, Marzie Mahdizade Ari, Marzie Mahdizade Ari, Amirhoushang Alvandi, Amirhoushang Alvandi, Ramin Abiri, Ramin Abiri   +7 more
doaj   +1 more source

Nano Approaches to Nucleic Acid Delivery: Barriers, Solutions, and Current Landscape

WIREs Nanomedicine and Nanobiotechnology, Volume 17, Issue 2, March/April 2025.
Nano approaches for breaking barriers to nucleic acid delivery. ABSTRACT Nucleic acid (NA) therapy holds tremendous potential for treating a wide range of genetic diseases by the delivery of therapeutic genes into target cells. However, significant challenges exist in safely and effectively delivering these genes to their intended locations.
Joan Castaneda Gonzalez, Ki Wan Park, Dallin Brian Evans, Rishi Sharma, Om Sahaym, Shamila Gopalakrishnan, Aqib Iqbal dar, Tulio A. Valdez, Anjali Sharma   +8 more
wiley   +1 more source

Implications of siRNA Therapy in Bone Health: Silencing Communicates

Biomedicines
The global statistics of bone disorders, skeletal defects, and fractures are frightening. Several therapeutic strategies are being used to fix them; however, RNAi-based siRNA therapy is starting to prove to be a promising approach for the prevention of ...
Puneetpal Singh, Monica Singh, Baani Singh, Kirti Sharma, Nitin Kumar, Deepinder Singh, Harpal Singh Klair, Sarabjit Mastana   +7 more
doaj   +1 more source

Development, opportunities, and challenges of siRNA nucleic acid drugs

Molecular Therapy: Nucleic Acids
Small interfering RNA (siRNA) drugs were first proposed in 1999. They have reached the market for administration to patients after more than 20 years of development.
Bowen Xiao, Shaopeng Wang, Yu Pan, Wenjun Zhi, Chensheng Gu, Tao Guo, Jiaqi Zhai, Chenxu Li, Yong Q. Chen, Rong Wang   +9 more
doaj   +1 more source

RNA interference therapeutics for disease-associated proteins: from molecular design to clinical translation

Discover Medicine
RNA interference (RNAi) has emerged as a revolutionary therapeutic approach for silencing disease-associated genes with unprecedented specificity and precision.
Sarfaraz K. Niazi
doaj   +1 more source

The Risks of miRNA Therapeutics: In a Drug Target Perspective

Drug Design, Development and Therapy, 2021
Song Zhang, 1, 2 Zhujun Cheng, 3 Yanan Wang, 1 Tianyu Han 1 1Jiangxi Institute of Respiratory Disease, The First Affiliated Hospital of Nanchang University, Nanchang, 330006, People’s Republic of China; 2College of Pharmaceutical Sciences ...
Zhang S, Cheng Z, Wang Y, Han T
doaj  

RNA interference therapy in cardiology: will new targets improve therapeutic goals?

Drugs in Context
The discovery of RNA interference in 1998 opened avenues for the manipulation of gene expression, leading to the development of small interfering RNA (siRNA) drugs.
Renata TJ Fazoli, Luciano F Drager, Roberto Kalil-Filho, Giuliano Generoso   +3 more
doaj   +1 more source

Acute intermittent porphyria in a resource-limited setting: diagnostic pitfalls and emerging therapeutic perspectives

Journal of Rare Diseases
Background Acute intermittent porphyria (AIP) is a rare metabolic disorder caused by deficiency of hydroxymethylbilane synthase (HMBS), leading to accumulation of neurotoxic heme precursors. Its protean neurovisceral manifestations and the possibility of
Bhupender Arya, Pyrus Bhellum, Rajat Goyal, Avishek Sarangal, Gopal Krishana Bohra   +4 more
doaj   +1 more source

UEG Week 2025 Poster Presentations

United European Gastroenterology Journal, Volume 13, Issue S8, Page S803-S1476, October 2025.
wiley   +1 more source