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Penn study sheds light on genetic cause of early hearing loss

From the moment a baby is born, they are put through a battery of screenings to test for all sorts of characteristics, which includes the sense of hearing.

26 Oct 2017

New enzyme may one day enable genome surgery that erases harmful mutations

DNA editing just got a sharp, new pencil. Researchers have built an enzyme that can perform a previously impossible DNA swap, directly changing the DNA base pair from an A•T to a G•C.

26 Oct 2017

Timing of DNA damage within checkpoints matters to a cell's fate, study reports

DNA damage occurs routinely within your cells due to sun exposure, smoking and sometimes during the normal process of making new DNA.

26 Oct 2017

CRISPR - a genetic editor - used to edit DNA and RNA in two parallel studies

Two different studies have shown that CRISPR - a gene editing tool, could be the next big thing in manipulating and modifying the DNA to suit the needs.

26 Oct 2017

Single infusion of wildtype HSPCs halts cellular damage caused by neuromuscular disorder in mice

Researchers at University of California San Diego School of Medicine report that a single infusion of wildtype hematopoietic stem and progenitor cells (HSPCs) into a mouse model of Friedreich's ataxia (FA) measurably halted cellular damage caused by the degenerative disease.

25 Oct 2017

Scientists discover how immune cells clear the way for nerve regeneration after injury

Immune cells are normally associated with fighting infection but in a new study, scientists have discovered how they also help the nervous system clear debris, clearing the way for nerve regeneration after injury.

25 Oct 2017

Researchers find rare genetic variants that may help explain longevity in some CF patients

Cystic fibrosis is an inherited disorder caused by genetic mutations that disrupt the normal movement of chloride in and out of cells. Among other health problems, cystic fibrosis compromises the lungs' ability to fight infection and breathe efficiently, making it the most lethal genetic disease in the Caucasian population. Patients have an average lifespan of just 30 to 40 years.

25 Oct 2017

Insights from NGLY1 deficiency could provide new approach to treat blood cancers

A new class of drugs for blood cancers such as leukemia and multiple myeloma is showing promise. But it is hobbled by a problem that also plagues other cancer drugs: targeted cells can develop resistance.

25 Oct 2017

'Intelligent' nanoparticles could help treat cancer patients

Scientists from the University of Surrey have developed 'intelligent' nanoparticles which heat up to a temperature high enough to kill cancerous cells - but which then self-regulate and lose heat before they get hot enough to harm healthy tissue.

24 Oct 2017

Study finds lung cancer driver ALK-fusion in mucosal melanoma

Melanomas caused by sun exposure have been matched with targeted treatments and immunotherapies, in many cases dramatically extending patients' lives. However, there are other kinds of melanoma not related to sun exposure and because they are caused by different genetic changes, they are not susceptible to the same targeted treatments.

23 Oct 2017

Penn researchers provide new insights into how Notch drives growth of B-cell cancers

Notch is one of the most frequently mutated genes in chronic lymphocytic leukemia (CLL), the most common leukemia in adults in the United States. It is also often mutated in other common B cell tumors, such as mantle cell lymphoma.

23 Oct 2017

New study explores how herpes simplex virus may change during familial transmission

A new study explores how herpes simplex virus might change when passed from one individual to another, information that may prove useful in future development of therapeutics and vaccines.

20 Oct 2017

UC San Diego researchers develop 3D visualization tool for mapping omics data onto organs

University of California San Diego researchers have developed the first 3D spatial visualization tool for mapping "'omics" data onto whole organs.

20 Oct 2017

Penn researchers functionally dissect gene variant linked to FTLD

Seven years ago, Penn Medicine researchers showed that mutations in the TMEM106B gene significantly increased a person's risk of frontotemporal lobar degeneration (FTLD), the second most common cause of dementia in those under 65.

19 Oct 2017

FDA advisory committee vote 16-0 for first even gene therapy for rare blindness

A panel of advisors to the United States Food and Drug Administration (FDA) has voted unanimously in favor of an experimental gene therapy to treat patients with a rare kind of hereditary blindness.

16 Oct 2017

Dietary supplement could be promising therapeutic target for seizure disorders

Seizure disorders -- including epilepsy -- are associated with pathological hyperexcitability in brain neurons. Unfortunately, there are limited available treatments that can prevent this hyperexcitability.

13 Oct 2017

TGen awarded $2.5 million SU2C grant to test new method of treating pancreatic cancer

Stand Up To Cancer has awarded a $2.5 million SU2C CatalystR grant to the Translational Genomics Research Institute to investigate a revolutionary new method of treating pancreatic cancer, one of the most aggressive of all cancers.

13 Oct 2017

TSRI scientists develop molecular model for improving design of osteoporosis treatments

Using advanced mass spectrometry technology, scientists from the Florida campus of The Scripps Research Institute have developed a molecular model that may provide a new framework for improving the design of osteoporosis treatments.

13 Oct 2017

Study provides detailed characterization of neurotoxicity arising from CD19 CAR T-cell therapy

New potential biomarkers and a novel algorithm could help identify patients at increased risk of suffering from severe neurotoxicity after receiving CD19 CAR T-cell therapy. The study extensively characterized common and occasionally fatal side effects of this immunotherapy.

12 Oct 2017

Fundamental discovery may open potential for new types of cancer drugs

Many approved cancer therapies target a protein called epidermal growth factor receptor (EGFR) that regulates many crucial cellular processes and can speed the proliferation of tumor cells.

11 Oct 2017

Therapeutics News and Research

Penn study sheds light on genetic cause of early hearing loss

New enzyme may one day enable genome surgery that erases harmful mutations

Timing of DNA damage within checkpoints matters to a cell's fate, study reports

CRISPR - a genetic editor - used to edit DNA and RNA in two parallel studies

Single infusion of wildtype HSPCs halts cellular damage caused by neuromuscular disorder in mice

Scientists discover how immune cells clear the way for nerve regeneration after injury

Researchers find rare genetic variants that may help explain longevity in some CF patients

Insights from NGLY1 deficiency could provide new approach to treat blood cancers

'Intelligent' nanoparticles could help treat cancer patients

Study finds lung cancer driver ALK-fusion in mucosal melanoma

Penn researchers provide new insights into how Notch drives growth of B-cell cancers

New study explores how herpes simplex virus may change during familial transmission

UC San Diego researchers develop 3D visualization tool for mapping omics data onto organs

Penn researchers functionally dissect gene variant linked to FTLD

FDA advisory committee vote 16-0 for first even gene therapy for rare blindness

Dietary supplement could be promising therapeutic target for seizure disorders

TGen awarded $2.5 million SU2C grant to test new method of treating pancreatic cancer

TSRI scientists develop molecular model for improving design of osteoporosis treatments

Study provides detailed characterization of neurotoxicity arising from CD19 CAR T-cell therapy

Fundamental discovery may open potential for new types of cancer drugs

Advancing therapeutic biopolymer development eBook

Bringing real-time analytics to downstream bioprocess monitoring

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

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