𝔖 Bobbio Scriptorium
✦   LIBER   ✦

Tyrosinemia type 1 — complex splicing defects and a missense mutation in the fumarylacetoacetase gene

✍ Scribed by Helge Rootwelt; Tom Kristensen; Ruud Berger; Kari Høie; Eli Anne Kvittingen


Book ID
104665037
Publisher
Springer
Year
1994
Tongue
English
Weight
606 KB
Volume
94
Category
Article
ISSN
0340-6717

No coin nor oath required. For personal study only.

✦ Synopsis


Two mutations are reported in six tyrosinemia type 1 patients from northern Europe. In four patients, a G to A transition at nucleotide position 1009 (G1009-->A) of the fumarylacetoacetase (FAH) coding sequence caused aberrant splicing by introducing an acceptor splice site within exon 12, thereby deleting the first 50 nucleotides of this exon. The following exon-intron boundary was frequently missed, and a cryptic donor splice site within intron 12 caused a partial intron 12 retention of 105 bp. This point mutation alternatively gave a glycine 337 to serine substitution in instances of correct splicing. The mutation is rapidly detected by PvuII digestion of polymerase chain reaction (PCR)-amplified genomic DNA. Another mutation, g+5-->a in the intron 12 donor splice site consensus sequence (IVS12 g+5-->a), was found in five of the patients. This caused alternative splicing with retention of the first 105 nucleotides of intron 12, exon 12 skipping, and a combined deletion of exons 12 and 13. Rapid detection of this mutation is achieved by restriction digestion of PCR-amplified genomic DNA; a mismatch primer combined with the point mutation creates a Tru9I restriction site. One patient who was homozygous for the G1009-->A mutation had a chronic form of tyrosinemia. Three patients were combined heterozygotes for G1009-->A and IVS12 g+5-->a. Their clinical phenotypes varied from acute to chronic, indicating the impact of background genes and/or external factors on the presentation of tyrosinemia type 1.


📜 SIMILAR VOLUMES


Heterozygosity for an exon 12 splicing m
✍ Si Houn Hahn; Donna Krasnewich; Mark Brantly; Eli Anne Kvittingen; William A. Ga 📂 Article 📅 1995 🏛 John Wiley and Sons 🌐 English ⚖ 719 KB

Hereditary tyrosinemia type 1, an autosomal recessive disorder caused by deficiency of fumarylacetoacetate hydrolase (FAH), manifests in either an acute or a chronic form. We used reverse transcription and the polymerase chain reaction to amplify the FAH cDNA of a 12-year-old American boy with chron

The human fumarylacetoacetase gene: char
✍ Helge Rootwelt; Eli Anne Kvittingen; Kari Høie; Etienne Agsteribbe; Marijke Hart 📂 Article 📅 1992 🏛 Springer 🌐 English ⚖ 897 KB

Deficiency of human fumarylacetoacetase (FAH) activity results in hereditary tyrosinemia type I. Using the restriction enzymes BglII, KpnI and StuI and a 1.3-kb cDNA probe for the FAH gene, we have found 6 restriction fragment length polymorphisms (RFLPs). These RFLPs were utilised in 3 tyrosinemia

Germline mutations in the multiple endoc
✍ Matthew G. Mutch; William G. Dilley; Francisco Sanjurjo; Mary K. DeBenedetti; Ge 📂 Article 📅 1999 🏛 John Wiley and Sons 🌐 English ⚖ 463 KB 👁 1 views

Multiple endocrine neoplasia type 1 (MEN 1) is a familial cancer syndrome characterized by parathyroid hyperplasia, pituitary adenomas, and neuroendocrine tumors of the pancreas and duodenum. In 1997, the MEN1 tumor suppressor gene was identified, and numerous germline mutations have been reported t