STRUCTURE AND FUNCTION OF THE GROUP II INTRON RIBOZYME
Project Number5R01GM050313-04
Contact PI/Project LeaderPYLE, ANNA MARIE
Awardee OrganizationCOLUMBIA UNIVERSITY HEALTH SCIENCES
Description
Abstract Text
This project will elucidate the structure and mechanism of the Group II
intron, a catalytic RNA molecule (or ribozyme) which remains
enzymologically and structurally uncharacterized. Group II intron self-
splicing is central to the metabolism of higher plants and it shares
mechanistic features with the eukaryotic pre-mRNA splicing apparatus.
For this reason, it may constitute a model for the chemical and
structural elements involved in all eukaryotic RNA splicing. Because of
its similarities to our own forms of RNA processing, the Group II intron
may provide insight into the remnants of RNA catalysis which remains
important in higher organisms. The active site and substrate specificity
of the Group II intron appear different from those of other ribozymes,
so its characterization will push back the limits of known RNA reactivity
and increase the base of knowledge required for application of ribozymes
in gene therapy. The first objective of this study is to compare the
reactivity of known self-splicing Group II introns to learn which one is
most kinetically efficient and to understand how differences in Group II
morphology affect their reactivity. Efficiency of the introns in two-
step and single-step reactions will be compared by monitoring rates of
exon ligation and cleavage from 32p-labeled RNA transcripts. Based on
this study, a particular Group II intron will become the focus of further
investigation. The second objective is to determine the location of
tertiary interactions between catalytically essential Domains 1 and 5 of
the Group II intron. These interactions are unusual forms of RNA-RNA
contact potentially analogous to the ribose 2;-OH--base contacts I
identified in the Tetrahymena ribozyme. Techniques of in-vitro selection
and RNA footprinting will aid in the identification of critical 2'- OH,
phosphate and base functionalities which are required for proper tertiary
structure formation . The third objective is to convert the Group II
self-splicing RNA from a unimolecular species to a multiple-turnover
ribozyme suitable for detailed enzymological analysis of the first step
of splicing. It is the first step of Group II splicing which is
potentially the most unusual. The multiple-turnover ribozyme will be
created by fragmenting the intron into catalytically essential Domains
1 and 5, by transcription of a Domain 5-Domain 6 nucleophilic cofactor
and synthesis of a ribozyme "substrate" analogous to 5'exon-intron
boundary sequences. The multicomponent ribozyme will be analyzed under
conditions which will yield the individual rate and binding constants
descriptive of the active site and catalytic mechanism. The kinetic
framework will then be used to test models for Group II intron reactivity
through mutagenesis and functional group substitution on the individual
components. The framework will also facilitate a fourth objective: to
establish analogies between subdomains of the Group II intron and small
nuclear RNAs (snRNAs) of the eukaryotic splicing apparatus (the
spliceosome). U and U2 snRNAs will be inserted in place of Domain 5 and
a portion of Domain 6 in reaction of the multiple turnover construct.
Exchange in catalytic function would provide firm evidence for RNA
catalysis within the ribonucleoprotein spliceosome.
No Sub Projects information available for 5R01GM050313-04
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