GENE THERAPY FOR NEURODEGENERATION

We use state-of-the-art gene editing technologies to correct pathogenic  mutations

RESEARCH

Motivation

The progressive degeneration of the structure and function of the central nervous system, peripheral nervous system or musculoskeletal system is responsible for a broad range of diseases such as Parkinson's disease, Alzheimer's disease or Duchenne muscular dystrophy, which are incurable, debilitating and, ultimately, lethal. As the incidence and prevalence of these diseases increase with age, they will become major challenges for medicine and public health in future years.

Genome Editing

The genome editing revolution that occurred in the past 5 years has enabled the creation of technologies for editing the genome of living cells. Gene editing tools have proven to be effective for activating, repressing or knocking out genes, integrating DNA at target sites or even introducing single base modifications at specific sequences. The wide range of genomic modifications that can be accomplished with gene editing tools enables multiple strategies for treating human diseases.

Gene Delivery

Viral gene delivery systems can now safely and effectively carry nucleic acids to target cell populations within living organisms.  AAV vectors, in particular, possess low pathogenicity and have demonstrated efficacy in numerous clinical trials, including ones for hemophilia B, choroideremia, lipoprotein lipase deficiency, and Leber congenital amaurosis type II, the latter of which earned regulatory approval by the FDA in 2017.

Our Goal

Gene editing and gene delivery technologies have matured independently and are now primed to be applied as a cohesive suite of molecular tools for providing novel therapeutic solutions to previously incurable human diseases. Our laboratory utilizes gene editing tools to develop treatments for some of the most lethal neurodegenerative and musculoskeletal diseases.

Approach
Diseases we target

PUBLICATIONS

CELL DISCOVERY
Molecular Therapy
GENOME BIOLOGY

COMPLETE PUBLICATION LIST

TEAM
Pablo Perez-Pinera M.D., Ph.D. Assistant Professor Department of Bioengineering Wendy Woods M.S. Lab Coordinator Department of Bioengineering

Pankaj Acharya Ph.D.

Postdoctoral Fellow

Department of Bioengineering

Michael Gapinske

Graduate Student

Department of Bioengineering

Jackson Winter

Graduate Student

Department of Bioengineering

Shraddha Shirguppe

Graduate Student

Department of Bioengineering

Kurt Kostan

Graduate Student

Department of Bioengineering

Eileen Johnson

Graduate Student

Department of Bioengineering

Past Members

Alexander Brown Ph.D.

 

Laurent Grant

 

Nathan Tague

 

Kate Love

 

Anton Christensen

 

Nikhil Shiva

 

Sony Manandhar

 

Micca Hecht

BIOE306: Biofabrication Fall Semester BioFabrication Lab is a course that provides hands on experiences relating to tissue engineering and design of synthetic biological machines. This laboratory course is structured to simulate a graduate experience where the students will design systems, run experiments, analyze data and report findings.
INSTRUCTION
ABOUT US
BIOE460: Gene Editing Lab Spring Semester The objective of this course is to provide the knowledge and hands-on experience required for both designing and building gene editing tools. This particular course will highlight diverse examples of therapeutic applications and it covers topics such as genome engineering, regulation of gene expression and synthetic life.
Department of Bioengineering University of Illinois at Urbana-Champaign 3252 Everitt Laboratory 1406 West Green Street Urbana, Illinois 61801 USA E-mail: pablo(a)illinois.edu

© 2015 Pablo Perez-Pinera.

RESEARCH
Approach
Diseases we target
CELL DISCOVERY
GENOME BIOLOGY
TEAM
Pablo Perez-Pinera M.D., Ph.D. Assistant Professor Department of Bioengineering Wendy Woods M.S. Lab Coordinator Department of Bioengineering
Past Members
BIOE306: Biofabrication Fall Semester BioFabrication Lab is a course that provides hands on experiences relating to tissue engineering and design of synthetic biological machines. This laboratory course is structured to simulate a graduate experience where the students will design systems, run experiments, analyze data and report findings.
INSTRUCTION ABOUT US
BIOE460: Gene Editing Lab Spring Semester The objective of this course is to provide the knowledge and hands-on experience required for both designing and building gene editing tools. This particular course will highlight diverse examples of therapeutic applications and it covers topics such as genome engineering, regulation of gene expression and synthetic life.
Department of Bioengineering University of Illinois at Urbana-Champaign 3252 Everitt Laboratory 1406 West Green Street Urbana, Illinois 61801 USA E-mail: pablo(a)illinois.edu
RESEARCH
Approach
Diseases we target
CELL DISCOVERY
GENOME BIOLOGY
TEAM
Pablo Perez-Pinera M.D., Ph.D. Assistant Professor Department of Bioengineering
Wendy Woods M.S. Lab Coordinator Department of Bioengineering
Past Members
BIOE306: Biofabrication Fall Semester BioFabrication Lab is a course that provides hands on experiences relating to tissue engineering and design of synthetic biological machines. This laboratory course is structured to simulate a graduate experience where the students will design systems, run experiments, analyze data and report findings.
INSTRUCTION
BIOE460: Gene Editing Lab Spring Semester The objective of this course is to provide the knowledge and hands-on experience required for both designing and building gene editing tools. This particular course will highlight diverse examples of therapeutic applications and it covers topics such as genome engineering, regulation of gene expression and synthetic life.
ABOUT US Department of Bioengineering University of Illinois at Urbana-Champaign 3252 Everitt Laboratory 1406 West Green Street Urbana, Illinois 61801 USA E-mail: pablo(a)illinois.edu