Hemophilia News and Research

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Hemophilia is a rare, inherited bleeding disorder in which your blood doesn’t clot normally. If you have hemophilia, you may bleed for a longer time than others after an injury. You also may bleed internally, especially in your knees, ankles, and elbows. This bleeding can damage your organs or tissues and, sometimes, be fatal.

Study provides clues to how cancer cells develop resistance to chemotherapies

About half of all drugs, ranging from morphine to penicillin, come from compounds that are from-;or have been derived from-;nature. This includes many cancer drugs, which are toxic enough to kill cancer cells.

22 Jun 2018

Chemists invent new method for remote chiral induction

Chemists at Scripps Research have addressed one of the most formidable challenges in synthetic chemistry by inventing a method for "enantioselective remote meta-CH activation," which enables the making of chiral molecules that were previously difficult or impossible to synthesize.

19 Jun 2018

Researchers use droplet-sized 'miniecosystems' to test therapeutic potential of molecules

Scripps Research scientists have solved a major problem in chemistry and drug development by using droplet-sized 'miniecosystems' to quickly see if a molecule can function as a potential therapeutic.

19 Jun 2018

Scientists could build better drugs by learning from bacteria-derived molecules

Bacteria found in soil may harbor a potential game-changer for drug design. A new study by Scripps Research, published today in Nature Communications, suggests scientists could build better drugs by learning from bacteria-derived molecules called thiocarboxylic acids.

19 Jun 2018

Study finds increased risk of mortality in people with cold agglutinin disease in five years after diagnosis

Bioverativ Inc., a Sanofi company dedicated to transforming the lives of people with rare blood disorders, today announced results from a new, population-based retrospective study that found a greater than twofold increased risk of mortality in people with cold agglutinin disease within the first five years after diagnosis.

18 Jun 2018

Scientists engineer bi-specific antibodies that attract immune killers directly to cancer cells

Our immune system's arsenal of defenses usually protects us from cancer. But sometimes, cancer cells overwhelm or evade this elaborate defense system.

30 May 2018

New RNA deletion tool could help create drugs that correct genetic diseases

As scientists gain insights into which genes drive diseases, they are pursuing the next logical question: Can gene editing technologies be developed to treat or even cure those diseases? Much of that effort has focused on developing technologies such as CRISPR-Cas9, a protein-based system.

30 May 2018

LogicBio team to present data on preclinical effectiveness of GeneRide platform

LogicBio Therapeutics, Inc., a genetic medicine company founded to develop safe medicines with lasting therapeutic benefit for children with life-threatening diseases, today announced it will be presenting data on the company's technology and robust preclinical pipeline at the annual meeting of the American Society of Gene & Cell Therapy at the Hilton Chicago, from May 16-19, 2018.

14 May 2018

Researchers devise neuronal cookbook for turning skin cells into different types of neurons

Scientists at The Scripps Research Institute have devised what they call a "neuronal cookbook" for turning skin cells into different types of neurons.

10 May 2018

Scripps Research professor wins 2018 RSC Bioorganic Chemistry Award

Floyd Romesberg, PhD, professor at The Scripps Research Institute, has won the 2018 Royal Society of Chemistry Bioorganic Chemistry Award for his ground-breaking contributions to the expansion of the genetic alphabet.

9 May 2018

Scientists discover how potential new drug targets spinal muscular atrophy

Spinal muscular atrophy (SMA) is a genetic disease that can leave infants with weak muscles and trouble breathing. Many with the disease die before age two. To help these patients, doctors need therapies that target the genetic mutation and stop its progression.

8 May 2018

Scripps Research team receives $12 million grant to improve flu and malaria vaccines

Scientists at The Scripps Research Institute aim to improve flu and malaria vaccines with the support of a new $12 million grant from the Bill & Melinda Gates Foundation.

3 May 2018

Scripps researchers awarded two grants to support studies on cocaine, oxycodone addiction

Last year, more than 60,000 Americans died of an opioid overdose, and millions more struggled with opioid and cocaine addiction.

27 Apr 2018

Breakthrough study promises to bring gene-editing technology much closer to therapeutic reality

Imagine a future where a guided biomachine put into your body seeks out defective gene sequences in each cell and edits in the correct information with precision accuracy.

13 Apr 2018

Scripps Florida researchers awarded $2.875 million to destroy common form of leukemia

Christoph Rader, PhD, associate professor at the Florida campus of The Scripps Research Institute, has been awarded a $2.875 million, five-year grant from the National Cancer Institute to develop unique antibody-drug conjugates engineered to eradicate one of the most common forms of leukemia, chronic lymphocytic leukemia

10 Apr 2018

Genetic mutation that may protect people from malaria found to be more common

A genetic mutation that may protect people from malaria, but was thought to be rare, is surprisingly common, suggest the findings of a new study led by scientists at The Scripps Research Institute.

23 Mar 2018

UCLA-developed nanospears could make gene therapies safer, faster and more cost-effective

UCLA scientists have developed a new method that utilizes microscopic splinter-like structures called "nanospears" for the targeted delivery of biomolecules such as genes straight to patient cells. These magnetically guided nanostructures could enable gene therapies that are safer, faster and more cost-effective.

15 Mar 2018

Hemophilia News and Research

Further Reading

Study provides clues to how cancer cells develop resistance to chemotherapies

Chemists invent new method for remote chiral induction

Researchers use droplet-sized 'miniecosystems' to test therapeutic potential of molecules

Scientists could build better drugs by learning from bacteria-derived molecules

Study finds increased risk of mortality in people with cold agglutinin disease in five years after diagnosis

Scientists engineer bi-specific antibodies that attract immune killers directly to cancer cells

New RNA deletion tool could help create drugs that correct genetic diseases

LogicBio team to present data on preclinical effectiveness of GeneRide platform

Researchers devise neuronal cookbook for turning skin cells into different types of neurons

Scripps Research professor wins 2018 RSC Bioorganic Chemistry Award

Scientists discover how potential new drug targets spinal muscular atrophy

Scripps Research team receives $12 million grant to improve flu and malaria vaccines

Scripps researchers awarded two grants to support studies on cocaine, oxycodone addiction

Breakthrough study promises to bring gene-editing technology much closer to therapeutic reality

Scripps Florida researchers awarded $2.875 million to destroy common form of leukemia

Genetic mutation that may protect people from malaria found to be more common

UCLA-developed nanospears could make gene therapies safer, faster and more cost-effective

TSRI scientists discover new pathway to promote mitochondrial function during stress

Of ‘miracles’ and money: Why hemophilia drugs are so expensive

TSRI research offers explanation for why neurons die off in Parkinson's disease

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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