KU News: KU, partners receive $1.2M to transform understanding of RNA splicing

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KU, partners receive $1.2M to transform understanding of RNA splicing
LAWRENCE — The W.M. Keck Foundation has awarded a $1.2 million grant to medicinal chemistry and computational biology researchers at the University of Kansas and the University of Chicago focused on addressing a long-unresolved problem in biomedical research — finding molecules able to target the “undruggable proteome.”

KU launches Higher Learning Commission accreditation reaffirmation process
LAWRENCE — In a little more than two years, the University of Kansas will undergo evaluation by the Higher Learning Commission (HLC) to reaffirm its accreditation. On Aug. 29, steering committee team members from all KU campuses will assemble in the Kansas Union’s Big 12 Room to formally begin a comprehensive process.

Full stories below.

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Contact: Brad Stauffer, School of Pharmacy, [email protected], @KUPharmacy
KU, partners receive $1.2M to transform understanding of RNA splicing
LAWRENCE — The W.M. Keck Foundation has awarded a $1.2 million grant to medicinal chemistry and computational biology researchers at the University of Kansas and the University of Chicago focused on addressing a long-unresolved problem in biomedical research — finding molecules able to target the “undruggable proteome.”
Jingxin Wang, assistant professor of medicinal chemistry at KU, applied for and received the Keck funding jointly with Yang Li, assistant professor of medicine and human genetics at the University of Chicago. Using RNA splicing modulators (a type of molecule) coupled with deep learning models (a subfield of artificial intelligence), their research holds promise to be a game-changer in drug discovery and disease research. The pair of researchers and their lab colleagues are working to identify human gene sequences that will respond to drug therapies through RNA splicing. It could open the door to successful new disease therapies and cures.
“Approximately 70% of the proteome (the entire set of proteins in certain human tissues) cannot be targeted by a drug,” Wang said. “This is alarming because we sometimes know how disease happens, but we don’t have any method to treat the disease. This is basically the undruggable proteome problem in medicinal chemistry.”
Wang and Li’s research seeks to address the undruggable problem. Recently, several RNA splicing modulators have been approved by the Food and Drug Administration for the life-saving treatment of previously untreatable spinal muscular atrophy and Duchenne muscular dystrophy.
“If we can precisely map where we can target RNA splicing, then researchers can focus on those genes and RNA sequences for drug development,” Wang said. “After systematic investigation, we will have a comprehensive map of splicing regulatory sequences for the whole human genome, and this will be a very valuable resource, not only for us but for the entire research community.”
The biochemical process in human cell development and replication is a complex system at the molecular level. Genes are encoded in DNA, which passes on the genetic information to RNA in a process called transcription. Then, RNA translates the encoded genetic information to protein in cells. The proteins usually act as final “executors” that perform or regulate most cell functions.
Wang’s lab is focusing on RNA splicing, an essential biological process in humans that happens before the final RNA is delivered to the protein. The processes of transcription, splicing and translation work together to dictate the amount and composition of proteins, which are drastically different among different tissues or cell states of health, development, disease and defense.
Wang’s lab at KU has an ambitious plan to focus on a subset of 100 genes to systematically identify the splicing regulatory sequence using chemical probes. To develop a map of druggable genes, Wang and Li propose taking the data from those experiments to train a deep learning model. Li’s lab at the University of Chicago will use machine learning to analyze and predict which of the 20,000 human genes are likely to respond to drugs that target RNA splicing.
“It’s quite a challenge to sift through that much data to identify which genes could be good targets,” Li said. “We are fortunate for this opportunity to collaborate with our partners to develop new computational approaches to study how targeting RNA splicing can overcome the ‘undruggable proteome’ problem.”
Wang said the Keck Foundation’s support is crucial to building a new platform of precision medicine in the treatment of disease.
“Without this Keck grant, we can’t gather or obtain those data,” Wang said, noting it would be necessary for agencies like the National Institutes of Health to fund additional research. “I’m so grateful that the Keck Foundation views research in a different way so that important projects like this are funded and get going. If successful, this will be one of the most advanced technologies in the field of RNA splicing.”
KU Endowment serves as a liaison with the Keck Foundation and was instrumental in supporting the pre-proposal and proposal development. University of Chicago Corporate and Foundation Relations also assisted in the grant proposal and presentation preparations.
Learn more about the W.M. Keck Foundation.
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Contact: Robin Lehman, Office of the Provost, 785-864-4410, [email protected], @KUProvost
KU launches Higher Learning Commission accreditation reaffirmation process

LAWRENCE — In a little more than two years, the University of Kansas will undergo evaluation by the Higher Learning Commission (HLC) to reaffirm its accreditation. On Aug. 29, steering committee team members from all KU campuses will assemble in the Kansas Union’s Big 12 Room to formally begin a comprehensive process.
There are three key initiatives related to KU’s work to prepare for reaffirmation:
1. The gathering of hundreds of artifacts that provide evidence KU not only meets but exceeds the core components of the HLC criteria and other requirements.
2. Institutionalizing accreditation to ensure accountability.
3. The adoption of a Quality Initiative (QI) aimed at a major improvement effort.
KU’s QI project, co-led by Neal Kingston, director of the Achievement & Assessment Institute, and Michelle Mohr Carney, dean of the School of Social Welfare, focuses on the adoption of universitywide institutional learning goals. It will work to strengthen and scale degree-level assessment at the Lawrence and Edwards campuses by developing and implementing degree maps that integrate the institutional learning goals and degree-level learning outcomes for all undergraduate and graduate programs. The QI project will also revise the academic program review processes at Lawrence and Edwards to achieve a stronger alignment of degree-level learning outcomes assessment with annual budgeting, operations and strategic plan implementation to create a culture of continuous quality improvement at KU. The Center for Teaching Excellence will also play a key role in the implementation of the QI project.
Along with ensuring continuous improvement, HLC accreditation is important because it is a requirement for awarding federal financial aid, which about half of all KU students currently receive. In addition, many academic programs need regional accreditation as a condition of receiving specialized accreditation. The Kansas Board of Regents also requires KU to maintain HLC accreditation in order to award degrees.
“As KU once again engages in a comprehensive self-study that ensures our continued institutional accreditation, it provides us with important opportunities to see where we’re performing well and areas that need improvement,” said Douglas A. Girod, KU’s chancellor. “It requires us to prove our case rather than state our case, demonstrating to our stakeholders that KU not only provides a quality educational experience but engages in continuous improvement across the core functions of our academic, research and operational missions.”
KU has been continuously accredited by the HLC since Jan. 1, 1913. The regional accrediting agency, recognized by the U.S. Department of Education, requires institutions under its authority to perform a comprehensive self-study every 10 years to demonstrate they meet the requirements for accreditation. Once an institution’s self-report is submitted, a peer review team is selected to conduct the evaluation, which includes visits to the university’s campuses and meetings with stakeholders. The reviewers then make a recommendation for continued accreditation.
HLC requires that the self-study process involve a diverse and representative cross-section of the university community. KU’s steering committee, which includes co-leads from the Lawrence and Edwards campuses and the Medical Center campus for each of the five criteria, reflects the diversity of the universitywide community of faculty, staff, students, governance representatives and administrators, and it will further represent a wide cross-section as members are added to the criterion subcommittees.
“The individuals who have agreed to serve in this critical capacity and take on the task of gathering a broad range of evidence deserve our gratitude and support as they conduct this work over the next 26 months,” Girod said.
KU’s self-study will be submitted to HLC in fall 2024. The peer review team is expected to visit campuses as part of its evaluation process early in 2025.

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