IDG

The Institute of Developmental Genetics (IDG) develops novel genetic tools and gene therapies. Using preclinical mouse and human cell models, it investigates the molecular causes of Parkinson’s disease to advance therapies like somatic cell reprogramming into neurons.

Correcting mutation by genetic engineering

The Institute of Developmental Genetics (IDG) focuses on the development of novel genetic tools (i.e. gene scissors) and the establishment of innovative gene therapeutical approaches. In addition, preclinical mouse and cellular human models are used to decipher the molecular underpinnings of the etiology of Parkinson´s Disease. Based on our findings therapeutic approaches, i.e. reprogramming of somatic cells to neurons, are developed and improved.

Genetic engineering and gene manipulation concept. Hand is replacing part of a DNA molecule.

Doctor holding in hand Regenerative Medicine

Laboratory transgenic mouse on the researcher`'s hand

Human (animal) cell under microscope. 3d illustration

Researchers at Helmholtz Munich received the pre-seed competition m4 Award 2025 for their project SYNTRA, which develops new methods to deliver RNA-based medicines using artificial intelligence (AI). The award supports innovative medical research in…

Picture with Cas13d-NCS for Press Release in Cell Discovery 2024

The rise of RNA viruses like SARS-CoV-2 highlights the need for new ways to fight them. RNA-targeting tools like CRISPR/Cas13 are powerful but inefficient in the cytoplasm of cells, where many RNA viruses replicate. Scientists from Helmholtz Munich…

Sporadic Parkinson's Disease (sPD) is a progressive neurodegenerative disorder influenced by both genetic and environmental factors. While mitochondrial dysfunction contributes to this condition, its role across disease progression remains unclear.…

Parkinson's disease, formerly known as shaking palsy, is the second most common neurodegenerative disease in Germany after Alzheimer's disease. It affects about 400,000 people in Germany alone and can neither be cured nor can its progression be…

Cover Art: The Cut and Restore Protein Trick: Self-Excising Designer Proteins Report Isoform Expression

Our proteome is much bigger than our genome because one gene produces several variants of proteins called protein isoforms, whose disbalance is implicated in many diseases. A new bioengineered reporter system developed at Helmholtz Zentrum München…

Reprogrammed neurons upon Ascl1 expression and neuron-enriched mitochondrial proteins.

The replacement of lost neurons is a holy grail for neuroscience. A new promising approach is the conversion of glial cells into new neurons. Improving the efficiency of this conversion or reprogramming after brain injury is an important step towards…

Duchenne type muscular dystrophy (DMD) is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of twelve and reducing life expectancy. Researchers at Helmholtz Zentrum München, Technical University…

Gruber C, Krautner L, Bergant V, Grass V, Ma Z, Rheinemann L, Krus A, Reinhardt F, Mazneykova L, Rocha-Hasler M, Truong DJ, Westmeyer GG, Pichlmair A, Ebert G, Giesert F, Wurst W.

Engineered, nucleocytoplasmic shuttling Cas13d enables highly efficient cytosolic RNA targeting.

Schmidt S, Stautner C, Vu DT, Heinz A, Regensburger M, Karayel O, Trümbach D, Artati A, Kaltenhäuser S, Nassef MZ, Hembach S, Steinert L, Winner B, Jürgen W, Jastroch M, Luecken MD, Theis FJ, Westmeyer GG, Adamski J, Mann M, Hiller K, Giesert F, Vogt Weisenhorn DM, Wurst W.

A reversible state of hypometabolism in a human cellular model of sporadic Parkinson's disease.

Schmidt S, Luecken MD, Trümbach D, Hembach S, Niedermeier KM, Wenck N, Pflügler K, Stautner C, Böttcher A, Lickert H, Ramirez-Suastegui C, Ahmad R, Ziller MJ, Fitzgerald JC, Ruf V, van de Berg WDJ, Jonker AJ, Gasser T, Winner B, Winkler J, Vogt Weisenhorn DM, Giesert F, Theis FJ, Wurst W.

Primary cilia and SHH signaling impairments in human and mouse models of Parkinson's disease.

Giehrl-Schwab J, Giesert F, Rauser B, Lao CL, Hembach S, Lefort S, Ibarra IL, Koupourtidou C, Luecken MD, Truong DJ, Fischer-Sternjak J, Masserdotti G, Prakash N, Ninkovic J, Hölter SM, Vogt Weisenhorn DM, Theis FJ, Götz M, Wurst W.

Parkinson's disease motor symptoms rescue by CRISPRa-reprogramming astrocytes into GABAergic neurons.

Russo GL, Sonsalla G, Natarajan P, Breunig CT, Bulli G, Merl-Pham J, Schmitt S, Giehrl-Schwab J, Giesert F, Jastroch M, Zischka H, Wurst W, Stricker SH, Hauck SM, Masserdotti G, Götz M.

CRISPR-Mediated Induction of Neuron-Enriched Mitochondrial Proteins Boosts Direct Glia-to-Neuron Conversion.

Moretti A, Fonteyne L, Giesert F, Hoppmann P, Meier AB, Bozoglu T, Baehr A, Schneider CM, Sinnecker D, Klett K, Fröhlich T, Rahman FA, Haufe T, Sun S, Jurisch V, Kessler B, Hinkel R, Dirschinger R, Martens E, Jilek C, Graf A, Krebs S, Santamaria G, Kurome M, Zakhartchenko V, Campbell B, Voelse K, Wolf A, Ziegler T, Reichert S, Lee S, Flenkenthaler F, Dorn T, Jeremias I, Blum H, Dendorfer A, Schnieke A, Krause S, Walter MC, Klymiuk N, Laugwitz KL, Wolf E, Wurst W, Kupatt C.

Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy.