Gene Therapy and Inborn Errors [GTIE] Special Emphasis Panel

(This is a recurring Special Emphasis Panel.)

[GTIE Roster]

Genetic basis of defects in lipid, amino acid, carbohydrate and nucleic acid metabolism and organelle function and development of strategies for their correction. This can include investigation of inborn errors of metabolism, mitochondrial defects, mechanism of mutation and gene silencing, replacement or repair. 

Specific areas covered by GTIE:

  • Development of gene therapy approaches for metabolic diseases, including lysosomal, peroxisomal and mitochondrial storage diseases, affecting multiple organs
  • Molecular genetics of viral and non-viral vectors within target cells and tissues:
  • Studies of transduction, integration, replication and repair, gene expression and gene silencing mechanisms in animal and human tissues and in animal models of diseases
  • Studies of inborn errors and other rare diseases: including biochemical genetics to elucidate regulation and dysregulation in metabolic pathways; studies of genetic mutations, transcriptional networks, protein structure/function and post-translational modifications; clinical manifestations; diagnosis and treatment development
  • Development of in vitro and animal models of disease for gene therapy investigation 

GTIE has the following shared interests within the GGG IRG: 

  • With Genetics of Health and Disease: GTIE and GHD both review applications dealing with the genetic basis of inborn errors of metabolism.  If the application is focused on gene identification, assignment could be to GHD.  If the focus is to understand molecular phenotypic effects of genetic alterations or developing gene therapy approaches for the disorder, assignment could go to GTIE.

  • With Molecular Genetics A, B, & C: MG study sections and GTIE review applications dealing with DNA replication repair, recombination and gene expression and silencing. Applications with a basic science context may be assigned to the MG study sections; applications with a disease or gene therapy context may be assigned to GTIE.

GTIE has the following shared interests outside the GGG IRG: 

  • With the Endocrinology, Metabolism, Nutrition & Reproductive Sciences [EMNR], Musculoskeletal, Oral, & Skin Sciences [MOSS], AIDS & Related Research [AARR], Oncological Sciences [ONC], Immunology [IMM], Infectious Diseases & Microbiology [IDM], Cardiovascular Sciences [CVS], Digestive Sciences [DIG], Hematology [HEME], Respiratory Sciences [RES], and Renal & Urological Sciences [RUS] IRGs with respect to gene therapy of metabolic diseases. If the application concerns a particular disease or organ system, assignment could be to the disease/organ IRG. If the application concerns issues of broad interest to the field of gene therapy, multiple organs or an emerging approach, assignment could be to GTIE.

  • With the Neuroscience IRGs - Brain Disorders & Clinical Neuroscience [BDCN], Integrative, Functional, & Cognitive Neuroscience [IFCN], and Molecular, Cellular, & Developmental Neuroscience[MDCN] in the study of metabolic disorders that affect the function of the nervous system. If neuropathology is the main focus, the application could be assigned to BDCN, IFCN, or MDCN, if a metabolic defect, issues of broad interest to the field of gene therapy, or an emerging genetic approach is the main focus, then assignment could be to GTIE.

  • With the Bioengineering Sciences & Technologies [BST] IRG with respect to gene and drug delivery systems.  If the focus is on bioengineering, development, design, or validation of drug delivery systems, the assignment could be to BST.  If the focus is on gene therapy, the assignment could be to GTIE.


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