A noncoding RNA modulator potentiates phenylalanine metabolism in mice
Abstract
Phenylketonuria provides a classic case of the benefit of newborn metabolic screening: It is a single-gene disease that can be detected at birth, and its neurological effects can be prevented by dietary therapy. Unfortunately, this is not always straightforward because the disease-causing mutations in phenylalanine hydroxylase vary between patients and affect the severity of the phenotype, such that some patients’ symptoms do not fully respond to the available interventions. Li et al . identified two long noncoding RNAs, one in mice and one in humans, that interact with phenylalanine hydroxylase and modulate its function (see the Perspective by Ben-Tov Perry and Ulitsky). Administration of modified RNAs mimicking their effects ameliorated the disease phenotype in mouse models of phenylketonuria. —YN
Document Details
- Document Type
- Pub Defense Publication
- Publication Date
- Aug 06, 2021
- Source ID
- 10.1126/science.aba4991
Entities
People
- Ania Muntau
- Chunlai Li
- Chunru Lin
- Cristian Coarfa
- David H Hawke
- Farès Namour
- François Feillet
- George A. Calin
- Heidi Hsiao
- Jean-louis Guéant
- Jianming Xu
- Jun Yao
- Ke Liang
- Kuang‐Lei Tsai
- Lan Liao
- Leng Han
- Liuqing Yang
- Manuel Schiff
- Mien-Chie Hung
- Nagireddy Putluri
- Nenad Blau
- Preethi H. Gunaratne
- Qingsong Hu
- Sergey D. Egranov
- Shuxing Zhang
- Sujash S. Chatterjee
- Tina K. Nguyen
- Vernon Reid Sutton
- Yajuan Li
- Yaohua Zhang
- Yinghong Pan
- Yi‐Chuan Li
- Youqiong Ye
- Zhao Zhang
- Zhen Xing
- Zhi Tan
Organizations
- American Association for Cancer Research
- Asia University
- Baylor College of Medicine
- Cancer Prevention and Research Institute of Texas
- Children's Hospital Zurich
- China Medical University
- Moscow Aviation Institute
- National Cancer Institute
- National Institutes of Health
- National Science Foundation
- The University of Texas MD Anderson Cancer Center
- United States Department of Defense
- University of Houston
- University of Lorraine
- University of Paris
- University of Texas Health Science Center at Houston
- University of Texas at Austin